Incidence and Mortality

Estimated new cases and deaths from pancreatic cancer in the United States in 2025:[1]

The incidence of pancreatic cancer has markedly increased over the past several decades. In the United States, it ranks as the fourth leading cause of cancer death in men and the third leading cause of cancer death in women.[1] Despite the high mortality rate associated with pancreatic cancer, its etiology is poorly understood.

Risk Factors

Risk factors for development of pancreatic cancer include:[2,3]

Anatomy

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Cancers of the pancreas are commonly identified by the site of involvement within the pancreas. Surgical approaches differ for masses in the head, body, tail, or uncinate process of the pancreas.

Clinical Features

Pancreatic cancer symptoms depend on the site of the tumor within the pancreas and the degree of tumor involvement.

In the early stages of pancreatic cancer, there are not many noticeable symptoms. As the cancer grows, symptoms may include:

Diagnostic and Staging Evaluation

Pancreatic cancer is difficult to detect and diagnose for the following reasons:

To appropriately treat pancreatic cancer, it is crucial to evaluate whether the cancer can be resected.

Prognosis and Survival

The primary factors that influence prognosis are:

Exocrine pancreatic cancer is rarely curable and has an overall survival (OS) rate of less than 6%.[9] Pancreatic cancer is associated with significant morbidity and mortality, and treatment decisions are complex. Management with a comprehensive multidisciplinary team should be considered.

The highest cure rate occurs when the tumor is truly localized to the pancreas; however, this stage of disease accounts for less than 20% of cases. For patients with localized disease and small cancers (<2 cm) with no lymph node metastases and no extension beyond the capsule of the pancreas, complete surgical resection is associated with an actuarial 5-year survival rate of 18% to 24%.[10][Level of evidence C1]

Surgical resection is the mainstay of curative treatment and provides a survival benefit in patients with small, localized pancreatic tumors, but it should be considered only alongside systemic therapy. Patients with unresectable, metastatic, or recurrent disease are unlikely to benefit from surgical resection.

Patients with any stage of pancreatic cancer are candidates for clinical trials because of the poor response to chemotherapy, radiation therapy, and surgery as conventionally used.

Information about ongoing clinical trials for pancreatic cancer is available from the NCI website.

Palliative Therapy

Palliation of symptoms may be achieved with conventional treatment (systemic chemotherapy).

Palliative measures that may improve quality of life without affecting OS include:[11,12]

AJCC Stage Groupings and TNM Definitions

Table 1. Definitions for Exocrine Pancreas TNM Stage 0a

Stage	TNM	Description	Illustration
T = primary tumor; N = regional lymph node; M = distant metastasis.
aReprinted with permission from AJCC: Exocrine Pancreas. In: Amin MB, Edge SB, Greene FL, et al., eds.: AJCC Cancer Staging Manual. 8th ed. New York, NY: Springer, 2017, pp. 337–47.
0	Tis, N0, M0	Tis = Carcinoma in situ. This includes high-grade pancreatic intraepithelial neoplasia (PanIn-3), intraductal papillary mucinous neoplasm with high-grade dysplasia, intraductal tubulopapillary neoplasm with high-grade dysplasia, and mucinous cystic neoplasm with high-grade dysplasia.	Enlarge
		N0 = No regional lymph node metastases.
		M0 = No distant metastasis.

Table 2. Definitions for Exocrine Pancreas TNM Stages IA and IBa

Stage	TNM	Description	Illustration
T = primary tumor; N = regional lymph node; M = distant metastasis.
aReprinted with permission from AJCC: Exocrine Pancreas. In: Amin MB, Edge SB, Greene FL, et al., eds.: AJCC Cancer Staging Manual. 8th ed. New York, NY: Springer, 2017, pp. 337–47.
IA	T1, N0, M0	T1 = Tumor ≤2 cm in greatest dimension.	Enlarge
		–T1a = Tumor ≤0.5 cm in greatest dimension.
		–T1b = Tumor >0.5 cm and <1 cm in greatest dimension.
		–T1c = Tumor 1–2 cm in greatest dimension.
		N0 = No regional lymph node metastases.
		M0 = No distant metastasis.
IB	T2, N0, M0	T2 = Tumor >2 cm and ≤4 cm in greatest dimension.
		N0 = No regional lymph node metastases.
		M0 = No distant metastasis.

Table 3. Definitions for Exocrine Pancreas TNM Stages IIA and IIBa

Stage	TNM	Description	Illustration
T = primary tumor; N = regional lymph node; M = distant metastasis.
aReprinted with permission from AJCC: Exocrine Pancreas. In: Amin MB, Edge SB, Greene FL, et al., eds.: AJCC Cancer Staging Manual. 8th ed. New York, NY: Springer, 2017, pp. 337–47.
IIA	T3, N0, M0	T3 = Tumor >4 cm in greatest dimension.	Enlarge
		N0 = No regional lymph node metastases.
		M0 = No distant metastasis.
IIB	T1, N1, M0	T1 = Tumor ≤2 cm in greatest dimension.	Enlarge
		–T1a = Tumor ≤0.5 cm in greatest dimension.
		–T1b = Tumor >0.5 cm and <1 cm in greatest dimension.
		–T1c = Tumor 1–2 cm in greatest dimension.
		N1 = Metastasis in one to three regional lymph nodes.
		M0 = No distant metastasis.
	T2, N1, M0	T2 = Tumor >2 cm and ≤4 cm in greatest dimension.
		N1 = Metastasis in one to three regional lymph nodes.
		M0 = No distant metastasis.
	T3, N1, M0	T3 = Tumor >4 cm in greatest dimension.
		N1 = Metastasis in one to three regional lymph nodes.
		M0 = No distant metastasis.

Table 4. Definitions for Exocrine Pancreas TNM Stage IIIa

Stage	TNM	Description	Illustration
T = primary tumor; N = regional lymph node; M = distant metastasis.
aReprinted with permission from AJCC: Exocrine Pancreas. In: Amin MB, Edge SB, Greene FL, et al., eds.: AJCC Cancer Staging Manual. 8th ed. New York, NY: Springer, 2017, pp. 337–47.
III	T1, N2, M0	T1 = Tumor ≤2 cm in greatest dimension.	Enlarge
		–T1a = Tumor ≤0.5 cm in greatest dimension.
		–T1b = Tumor >0.5 cm and <1 cm in greatest dimension.
		–T1c = Tumor 1–2 cm in greatest dimension.
		N2 = Metastasis in four or more regional lymph nodes.
		M0 = No distant metastasis.
	T2, N2, M0	T2 = Tumor >2 cm and ≤4 cm in greatest dimension.
		N2 = Metastasis in four or more regional lymph nodes.
		M0 = No distant metastasis.
	T3, N2, M0	T3 = Tumor >4 cm in greatest dimension.
		N2 = Metastasis in four or more regional lymph nodes.
		M0 = No distant metastasis.
	T4, Any N, M0	T4 = Tumor involves celiac axis, superior mesenteric artery, and/or common hepatic artery, regardless of size.
		NX = Regional lymph nodes cannot be assessed.
		N0 = No regional lymph node metastases.
		N1 = Metastasis in one to three regional lymph nodes.
		N2 = Metastasis in four or more regional lymph nodes.
		M0 = No distant metastasis.

Table 5. Definitions for Exocrine Pancreas TNM Stage IVa

Stage	TNM	Description	Illustration
T = primary tumor; N = regional lymph node; M = distant metastasis.
aReprinted with permission from AJCC: Exocrine Pancreas. In: Amin MB, Edge SB, Greene FL, et al., eds.: AJCC Cancer Staging Manual. 8th ed. New York, NY: Springer, 2017, pp. 337–47.
IV	Any T, Any N, M1	TX = Primary tumor cannot be assessed.	Enlarge
		T0 = No evidence of primary tumor.
		Tis = Carcinoma in situ. This includes high-grade pancreatic intraepithelial neoplasia (PanIn-3), intraductal papillary mucinous neoplasm with high-grade dysplasia, intraductal tubulopapillary neoplasm with high-grade dysplasia, and mucinous cystic neoplasm with high-grade dysplasia.
		T1 = Tumor ≤2 cm in greatest dimension.
		–T1a = Tumor ≤0.5 cm in greatest dimension.
		–T1b = Tumor >0.5 cm and <1 cm in greatest dimension.
		–T1c = Tumor 1–2 cm in greatest dimension.
		T2 = Tumor >2 cm and ≤4 cm in greatest dimension.
		T3 = Tumor >4 cm in greatest dimension.
		T4 = Tumor involves celiac axis, superior mesenteric artery, and/or common hepatic artery, regardless of size.
		NX = Regional lymph nodes cannot be assessed.
		N0 = No regional lymph node metastases.
		N1 = Metastasis in one to three regional lymph nodes.
		N2 = Metastasis in four or more regional lymph nodes.
		M1 = Distant metastasis.

Capecitabine and Fluorouracil Dosing

Treatment Options for Resectable or Borderline Resectable Pancreatic Cancer

Treatment options for resectable or borderline resectable pancreatic cancer include:

Palliative therapies can be considered in patients with any stage of disease. For more information, see the Palliative Therapy section.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Treatment Options for Locally Advanced Pancreatic Cancer

While locally advanced and metastatic pancreatic cancer are both incurable, their natural histories may be different. An autopsy series demonstrated that 30% of patients presenting with locally advanced disease died without evidence of distant metastases.[1][Level of evidence A1] Therefore, investigators have struggled to determine whether chemoradiation therapy for patients presenting with locally advanced disease is warranted.

Treatment options for locally advanced pancreatic cancer include:

Palliative therapies can be considered in patients with any stage of disease. For more information, see the Palliative Therapy section.

Evidence (chemoradiation therapy):

Three trials evaluated combined modality therapy versus radiation therapy alone.[16–18] The trials had substantial deficiencies in design or analysis. Initially, the standard of practice was to give chemoradiation therapy based on data from the first two studies. However, with the publication of the third study, standard practice changed to chemotherapy followed by chemoradiation in the absence of metastases.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Treatment Options for Metastatic or Recurrent Pancreatic Cancer

Treatment options for metastatic or recurrent pancreatic cancer include:

Palliative therapies can be considered in patients with any stage of disease. For more information, see the Palliative Therapy section.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of adult pancreatic cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Pancreatic Cancer Treatment are:

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Pancreatic Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/pancreatic/hp/pancreatic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389394]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s Email Us.

Pancreatic neuroendocrine tumors form in hormone-making cells (islet cells) of the pancreas.

The pancreas is a gland about 6 inches long that is shaped like a thin pear lying on its side. The wider end of the pancreas is called the head, the middle section is called the body, and the narrow end is called the tail. The pancreas lies behind the stomach and in front of the spine.

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There are two kinds of cells in the pancreas:

This summary discusses islet cell tumors of the endocrine pancreas. See the PDQ summary on Pancreatic Cancer Treatment for information on exocrine pancreatic cancer.

Pancreatic neuroendocrine tumors (NETs) may be benign (not cancer) or malignant (cancer). When pancreatic NETs are malignant, they are called pancreatic endocrine cancer or islet cell carcinoma.

Pancreatic NETs are much less common than pancreatic exocrine tumors and have a better prognosis.

Pancreatic NETs may or may not cause signs or symptoms.

Pancreatic NETs may be functional or nonfunctional:

Most pancreatic NETs are functional tumors.

There are different kinds of functional pancreatic NETs.

Pancreatic NETs make different kinds of hormones such as gastrin, insulin, and glucagon. Functional pancreatic NETs include the following:

Having certain syndromes can increase the risk of pancreatic NETs.

Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk.

Multiple endocrine neoplasia type 1 (MEN1) syndrome is a risk factor for pancreatic NETs.

Different types of pancreatic NETs have different signs and symptoms.

Signs or symptoms can be caused by the growth of the tumor and/or by hormones the tumor makes or by other conditions. Some tumors may not cause signs or symptoms. Check with your doctor if you have any of these problems.

Signs and symptoms of a non-functional pancreatic NET

A non-functional pancreatic NET may grow for a long time without causing signs or symptoms. It may grow large or spread to other parts of the body before it causes signs or symptoms, such as:

Signs and symptoms of a functional pancreatic NET

The signs and symptoms of a functional pancreatic NET depend on the type of hormone being made.

Too much gastrin may cause:

Too much insulin may cause:

Too much glucagon may cause:

Too much vasoactive intestinal peptide (VIP) may cause:

Too much somatostatin may cause:

A pancreatic NET may also make too much adrenocorticotropic hormone (ACTH) and cause Cushing syndrome. Signs and symptoms of Cushing syndrome include the following:

The treatment of pancreatic NETs that make too much ACTH and Cushing syndrome are not discussed in this summary.

Lab tests and imaging tests are used to diagnose pancreatic NETs.

The following tests and procedures may be used:

Other kinds of lab tests are used to check for the specific type of pancreatic NETs.

The following tests and procedures may be used:

Gastrinoma

Insulinoma

Glucagonoma

Other tumor types

Certain factors affect prognosis (chance of recovery) and treatment options.

Pancreatic NETs can often be cured. The prognosis and treatment options depend on the following:

The plan for cancer treatment depends on where the NET is found in the pancreas and whether it has spread.

The process used to find out if cancer has spread within the pancreas or to other parts of the body is called staging. The results of the tests and procedures used to diagnose pancreatic neuroendocrine tumors (NETs) are also used to find out whether the cancer has spread. See the General Information section for a description of these tests and procedures.

Although there is a standard staging system for pancreatic NETs, it is not used to plan treatment. Treatment of pancreatic NETs is based on the following:

There are three ways that cancer spreads in the body.

Cancer can spread through tissue, the lymph system, and the blood:

Cancer may spread from where it began to other parts of the body.

When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.

The metastatic tumor is the same type of tumor as the primary tumor. For example, if a pancreatic neuroendocrine tumor spreads to the liver, the tumor cells in the liver are actually neuroendocrine tumor cells. The disease is metastatic pancreatic neuroendocrine tumor, not liver cancer.

Pancreatic NETs can recur (come back) after they have been treated.

The tumors may come back in the pancreas or in other parts of the body.

There are different types of treatment for patients with pancreatic NETs.

Different types of treatments are available for patients with pancreatic neuroendocrine tumors (NETs). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The following types of treatment are used:

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI website.

Treatment for pancreatic neuroendocrine tumors may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today’s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).

To Learn More About Pancreatic Neuroendocrine Tumors (Islet Cell Tumors)

For more information from the National Cancer Institute about pancreatic neuroendocrine tumors (NETs), see the following:

For general cancer information and other resources from the National Cancer Institute, visit:

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute’s (NCI’s) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of pancreatic neuroendocrine tumors (islet cell tumors). It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

Reviewers and Updates

Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary (“Updated”) is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become “standard.” Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI’s website. For more information, call the Cancer Information Service (CIS), NCI’s contact center, at 1-800-4-CANCER (1-800-422-6237).

Permission to Use This Summary

PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].”

The best way to cite this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Pancreatic Neuroendocrine Tumors (Islet Cell Tumors) Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/pancreatic/patient/pnet-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389340]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.

Disclaimer

The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us.

Myelodysplastic/myeloproliferative neoplasms are a group of diseases in which the bone marrow makes too many white blood cells.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow.

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Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

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Myelodysplastic/myeloproliferative neoplasms have features of both myelodysplastic syndromes and myeloproliferative neoplasms.

In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets.

In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases.

This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. For more information about related diseases, see:

There are two main types of myelodysplastic/myeloproliferative neoplasms.

The two main types of myelodysplastic/myeloproliferative neoplasms in adults include:

When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC).

Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia.

Tests that examine the blood and bone marrow are used to diagnose myelodysplastic/myeloproliferative neoplasms.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

Chronic myelomonocytic leukemia is a disease in which too many myelocytes and monocytes (immature white blood cells) are made in the bone marrow.

In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.

Older age and being male increase the risk of chronic myelomonocytic leukemia.

Anything that increases a person’s chance of getting a disease is called a risk factor. Not every person with one or more of these risk factors will develop CMML, and it will develop in some people who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for CMML include:

Signs and symptoms of chronic myelomonocytic leukemia include fever, weight loss, and feeling very tired.

These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have:

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis and treatment options for CMML depend on:

Atypical chronic myeloid leukemia is a disease in which too many granulocytes (immature white blood cells) are made in the bone marrow.

In atypical chronic myeloid leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow.

The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the “Philadelphia chromosome,” is not there.

Signs and symptoms of atypical chronic myeloid leukemia include easy bruising or bleeding and feeling tired and weak.

These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have:

Certain factors affect prognosis (chance of recovery).

The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood.

Myelodysplastic/myeloproliferative neoplasm, unclassifiable, is a disease that has features of both myelodysplastic and myeloproliferative diseases but is not chronic myelomonocytic leukemia or atypical chronic myeloid leukemia.

In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets.

MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known.

Signs and symptoms of myelodysplastic/myeloproliferative neoplasm, unclassifiable, include fever, weight loss, and feeling very tired.

These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have:

There is no standard staging system for myelodysplastic/myeloproliferative neoplasms.

The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment.

There are different types of treatment for patients with myelodysplastic/myeloproliferative neoplasms.

Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The following types of treatment are used:

Stem cell transplant

Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.

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For more information, see Drugs Approved for Myeloproliferative Neoplasms or Myelodysplastic Syndromes.

New types of treatment are being tested in clinical trials.

For some people, joining a clinical trial may be an option. There are different types of clinical trials for people with cancer. For example, a treatment trial tests new treatments or new ways of using current treatments. Supportive care and palliative care trials look at ways to improve quality of life, especially for those who have side effects from cancer and its treatment.

You can use the clinical trial search to find NCI-supported cancer clinical trials accepting participants. The search allows you to filter trials based on the type of cancer, your age, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more about clinical trials, including how to find and join one, at Clinical Trials Information for Patients and Caregivers.

Treatment for myelodysplastic/myeloproliferative neoplasms may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Follow-up care may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute’s (NCI’s) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

Reviewers and Updates

Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary (“Updated”) is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become “standard.” Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI’s website. For more information, call the Cancer Information Service (CIS), NCI’s contact center, at 1-800-4-CANCER (1-800-422-6237).

Permission to Use This Summary

PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].”

The best way to cite this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.

Disclaimer

The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us.

Disease Overview

The myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are clonal myeloid disorders that have both dysplastic and proliferative features but are not properly classified as either myelodysplastic syndromes (MDS) or chronic myeloproliferative disorders (CMPD).[1–3] This category includes three major myeloid disorders: chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), and atypical chronic myeloid leukemia (aCML). Myeloid disease that shows features of both MDS and CMPD but does not meet the criteria for any of the three major MDS/MPN entities is designated as myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). The World Health Organization created the MDS/MPN category to provide a less restrictive view of myeloid disorders, which in some instances clearly overlap.[1–3]

Incidence and Mortality

The etiology of MDS/MPN is not known. The incidence of MDS/MPN varies widely, ranging from approximately 3 per 100,000 individuals older than 60 years annually for CMML to as few as 0.13 per 100,000 children from birth to 14 years annually for JMML.[4] Reliable data concerning the incidence of aCML, a recently defined entity, are not available. The incidence of MDS/MPN-UC is unknown.

Histopathology

The pathophysiology of MDS/MPN involves abnormalities in the regulation of myeloid pathways for cellular proliferation, maturation, and survival. Clinical symptoms result from the following complications:[5]

Patients with MDS/MPN do not have a Philadelphia chromosome or a BCR::ABL1 gene fusion.

An international consortium has proposed uniform response criteria to be used in clinical trials because of the spectrum of presentations ranging from the myelodysplastic to the myeloproliferative.[6]

Disease Overview

The World Health Organization (WHO) classifies chronic myelomonocytic leukemia (CMML) as a myelodysplastic/myeloproliferative neoplasm (MDS/MPN).[1] The WHO recognizes a dysplastic subtype and a proliferative subtype, with prognostic groups differentiated by the circulating white blood cell (WBC) count or the percentage of blasts in the bone marrow (higher percentage with worse prognosis).[2]

CMML is a clonal disorder of a bone marrow stem cell. Monocytosis is a major defining feature. CMML exhibits heterogenous clinical, hematological, and morphological features, varying from predominantly myelodysplastic to predominantly myeloproliferative. Evolution to acute myeloid leukemia (AML) portends a particularly poor prognosis.[3]

CMML is characterized pathologically by:[4,5]

Clinical features of CMML include:[4,5]

The median age at diagnosis of CMML is 65 to 75 years with a male predominance of 1.5 to 3.1.[4,5] Because CMML is grouped with chronic myeloid leukemia in some epidemiologic surveys and with MDS in others, no reliable incidence data are available for CMML.[6] Although the specific etiology of CMML is unknown, exposure to occupational and environmental carcinogens, ionizing radiation, and cytotoxic agents has been associated in some cases.[6]

Morphologically, the disease is characterized by a persistent peripheral blood monocytosis (always >1 × 109/L) that may exceed 80 × 109/L with monocytes typically accounting for more than 10% of the WBCs.[4,5] Monocytes, though typically mature with an unremarkable morphology, can exhibit abnormal granulation, unusual nuclear lobation, or finely dispersed nuclear chromatin.[7] Fewer than 20% blasts are seen in the blood or bone marrow. Neutrophilia occurs in nearly 50% of patients with neutrophil precursors (e.g., promyelocytes and myelocytes) accounting for more than 10% of the WBCs.[8] Mild normocytic anemia is common. Moderate thrombocytopenia is often present. Bone marrow findings include:[4,5,9,10]

Hepatosplenomegaly may be present.[4,5] Autoimmune phenomena, including pyoderma gangrenosum, vasculitis, and idiopathic thrombocytopenia have been observed in CMML.[11] Care should be taken to identify cases of CMML with eosinophilia, a subtype of CMML, because of its association with severe tissue damage secondary to eosinophil degranulation. In CMML with eosinophilia, all criteria for CMML are present, and the eosinophil count in the peripheral blood is more than 1.5 × 109/L.[6]

Recurrent somatic pathogenic variants have been identified in most patients with CMML, resulting in altered signaling molecules (especially NRAS, KRAS, JAK2, and SETBP1), epigenetic regulators (especially TET2 and ASXL1), splicing factors (especially SRSF2), and transcription factors (especially RUNX1).[12–15] A CMML-specific prognostic scoring system can distinguish four risk groups based on the following factors:[16]

The best prognostic group has a median survival of more than 10 years with no leukemic evolution in the first decade of follow-up. The worst prognostic group has a median survival of 20 months with a 50% evolution to AML by 2 years.[16]

Prognostic factors associated with shorter survival include:[17,18]

Progression to acute leukemia occurs in approximately 15% to 20% of cases.[17,18]

CPSS-Mol is a CMML-specific prognostic scoring system that incorporates molecular genetic data, especially pathogenic variants in RUNX1, NRAS, SETBP1, and ASXL1. This system distinguishes low-risk disease with median survivals longer than 10 years from high-risk disease with median survivals of 2 to 4 years.[16]

Treatment Overview

CMML is a diagnosis typically made after age 70 years. The clinical course of CMML ranges from indolent or smoldering disease to an aggressive disease progression culminating in severe cytopenias or evolution to acute leukemia. Assessment of the risk factors and the pace of disease over time may help to distinguish patients who require therapy from those who would be best managed with a watchful waiting approach. Asymptomatic patients at low risk of progression may be best served by forgoing therapy.[19,20]

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Disease Overview

Atypical chronic myeloid leukemia (aCML) is a leukemic disorder that exhibits both myelodysplastic and myeloproliferative features at the time of diagnosis.

Atypical CML is characterized pathologically by:[1]

Clinical features of aCML include:[1–4]

Although cytogenetic abnormalities are found in as many as 80% of the patients with aCML, none is specific.[1–3,5] No Philadelphia chromosome or BCR::ABL1 gene fusion is present.

The exact incidence of aCML is unknown. The median age at the time of diagnosis of this rare leukemic disorder is in the seventh or eighth decade of life.[1–3]

Morphologically, aCML is characterized by myelodysplasia associated with bone marrow and peripheral blood patterns similar to chronic myeloid leukemia, but cytogenetically it lacks a Philadelphia chromosome or BCR::ABL1 gene fusion.[1] The white blood cell count in the peripheral blood is variable. Median values range from 35 × 109/L to 96 × 109/L, and some patients may have white blood cell counts greater than 300 × 109/L.[1–3,5] Blasts in the peripheral blood typically account for less than 5% of the white blood cells. Immature neutrophils usually total 10% to 20% or more.[1] The percentage of monocytes is rarely more than 10%. Minimal basophilia may be present.[1–3,5] Nuclear abnormalities, such as acquired Pelger-Huët anomaly, may be seen in the neutrophils. Moderate anemia (often showing changes indicative of dyserythropoiesis) and thrombocytopenia are common.[1–4] Bone marrow findings include: [1–3,5]

The median survival times for aCML are reported to be less than 20 months, and thrombocytopenia and marked anemia are poor prognostic factors.[1,2] Atypical CML evolves to acute leukemia in approximately 25% to 40% of patients.[1,3] In the remaining patients, fatal complications include resistant leukocytosis, anemia, thrombocytopenia, hepatosplenomegaly, cerebral bleeding associated with thrombocytopenia, and infection.[3,4]

Treatment Overview

The optimal treatment of aCML is uncertain because of the rare incidence of this chronic leukemic disorder. Treatment with hydroxyurea may lead to short-lived partial remissions of 2 to 4 months in duration.[4] Atypical CML appears to respond poorly to treatment with interferon alfa.[4]

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Disease Overview

Myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) (also known as mixed myeloproliferative/myelodysplastic syndrome, unclassifiable and overlap syndrome, unclassifiable) shows features of both myeloproliferative disease and myelodysplastic disease but does not meet the criteria for any of the other MDS/MPN entities.[1]

Diagnostic criteria for MDS/MPN-UC can be either:[1]

The incidence and etiology of MDS/MPN-UC are unknown.

Laboratory features typically include anemia and dimorphic erythrocytes on the peripheral blood smear.[1] Thrombocytosis (platelet count >600 × 109/L) or leukocytosis (white blood cell count >13 × 109/L) are present. Neutrophils may exhibit dysplastic features, and giant or hypogranular platelets may be present. Blasts make up less than 20% of the white blood cells and of the nucleated cells of the bone marrow. The bone marrow is hypercellular and may exhibit proliferation in any or all of the myeloid lineages. Dysplastic features are present in at least one cell line.[1]

No cytogenetic or molecular findings are available that are specific for MDS/MPN-UC. In one small series, six of nine patients (those with ringed sideroblasts associated with marked thrombocytosis [RARS-T]) had JAK2 V617F variants, which caused constitutive activation of the JAK2 tyrosine kinase. This JAK2 pathogenic variant is also commonly observed in patients with polycythemia vera, essential thrombocythemia, and idiopathic myelofibrosis.[2] Because of its rare occurrence, the prognosis and predictive factors are unknown.[1]

Treatment Overview

Adult patients with MDS/MPN associated with platelet-derived growth factor receptor gene rearrangements are candidates for imatinib mesylate at standard dosages.[3] Because of its rare occurrence, the literature only minimally addresses other treatment options for MDS/MPN-UC. Supportive care involves treating cytopenias and infection as necessary.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of myelodysplastic/myeloproliferative neoplasms. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Myelodysplastic/Myeloproliferative Neoplasms Treatment are:

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/myeloproliferative/hp/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389321]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s Email Us.

Myelodysplastic syndromes are a group of cancers in which immature blood cells in the bone marrow do not mature or become healthy blood cells.

In a healthy person, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time.

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A blood stem cell may become a lymphoid stem cell or a myeloid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

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In a patient with a myelodysplastic syndrome, the blood stem cells (immature cells) do not become mature red blood cells, white blood cells, or platelets in the bone marrow. These immature blood cells, called blasts, do not work the way they should and either die in the bone marrow or soon after they go into the blood. This leaves less room for healthy white blood cells, red blood cells, and platelets to form in the bone marrow. When there are fewer healthy blood cells, infection, anemia, or easy bleeding may occur.

The different types of myelodysplastic syndromes are diagnosed based on certain changes in the blood cells and bone marrow.

Age and past treatment with chemotherapy or radiation therapy affect the risk of a myelodysplastic syndrome.

Anything that increases a person’s chance of getting a disease is called a risk factor. Not every person with one or more of these risk factors will develop myelodysplastic syndromes, and they will develop in people who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for myelodysplastic syndromes include the following:

The cause of myelodysplastic syndromes in most patients is not known.

Signs and symptoms of a myelodysplastic syndrome include shortness of breath and feeling tired.

Myelodysplastic syndromes often do not cause early signs or symptoms. They may be found during a routine blood test. Signs and symptoms may be caused by myelodysplastic syndromes or by other conditions. Check with your doctor if you have any of the following:

Tests that examine the blood and bone marrow are used to diagnose myelodysplastic syndromes.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis and treatment options depend on the following:

There are different types of treatment for patients with myelodysplastic syndromes.

Different types of treatment are available for patients with myelodysplastic syndromes. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Treatment for myelodysplastic syndromes includes supportive care, drug therapy, and stem cell transplant.

Patients with a myelodysplastic syndrome who have symptoms caused by low blood counts are given supportive care to relieve symptoms and improve quality of life. Drug therapy may be used to slow progression of the disease. Certain patients can be cured with aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.

The following types of treatment are used:

Chemotherapy with stem cell transplant

Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.

This treatment may not work as well in patients whose myelodysplastic syndrome was caused by past treatment for cancer.

Enlarge

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI website.

Treatment for myelodysplastic syndromes may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today’s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute’s (NCI’s) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of myelodysplastic syndromes. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

Reviewers and Updates

Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary (“Updated”) is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become “standard.” Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI’s website. For more information, call the Cancer Information Service (CIS), NCI’s contact center, at 1-800-4-CANCER (1-800-422-6237).

Permission to Use This Summary

PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].”

The best way to cite this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic Syndromes Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/myeloproliferative/patient/myelodysplastic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389239]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.

Disclaimer

The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us.

Incidence and Mortality

The MDS are a collection of myeloid malignancies characterized by one or more peripheral blood cytopenias. MDS are diagnosed in slightly more than 10,000 people in the United States yearly, for an annual age-adjusted incidence rate of approximately 4.4 to 4.6 cases per 100,000 people.[1] They are more common in men and White individuals. The syndromes may arise de novo or secondarily after treatment with chemotherapy and/or radiation therapy for other cancers or, rarely, after environmental exposures.

Prognosis

Prognosis is directly related to the number of bone marrow blast cells, to certain cytogenetic abnormalities, and to the amount of peripheral blood cytopenias. By convention, MDS are reclassified as acute myeloid leukemia (AML) with myelodysplastic features when blood or bone marrow blasts reach or exceed 20%. Many patients succumb to complications of cytopenias before progression to this stage. For more information, see the Pathological and Prognostic Systems for MDS section. The acute leukemic phase is less responsive to chemotherapy than is de novo AML.

Pathology

MDS are characterized by abnormal bone marrow and blood cell morphology. Megaloblastoid erythroid hyperplasia with macrocytic anemia, associated with normal vitamin B₁₂ and folate levels, is frequently observed. Circulating granulocytes are often hypogranular or hypergranular and may display the acquired pseudo-Pelger-Huët abnormality. Early, abnormal myeloid progenitors are identified in the marrow in varying percentages. Abnormally small megakaryocytes (micromegakaryocytes) may be seen in the marrow, and hypogranular or giant platelets may appear in the blood.

Clinical Features

MDS occur predominantly in older patients (usually older than 60 years), with a median age at diagnosis of approximately 70 years,[2] although patients as young as 2 years have been reported.[3] Anemia, bleeding, easy bruising, and fatigue are common initial findings. For more information, see Fatigue. Splenomegaly or hepatosplenomegaly may indicate an overlapping myeloproliferative neoplasm. Approximately 50% of patients have a detectable cytogenetic abnormality, most commonly a deletion of all or part of chromosome 5 or 7, or trisomy 8. Single-nucleotide polymorphism array technology may increase the detection of genetic abnormalities to 80%.[4,5] Although the bone marrow is usually hypercellular at diagnosis, 10% of patients present with a hypoplastic bone marrow.[6] Hypoplastic myelodysplastic patients tend to have profound cytopenias and may respond more frequently to immunosuppressive therapy.

Risk Factors

Approximately 90% of MDS cases occur de novo with no identifiable cause. Potential environmental risk factors for developing MDS include exposure to:[7,8]

Pathological Systems

The World Health Organization (WHO) classification [2] has supplanted the historic French-American-British (FAB) classification,[1] as shown in Table 1.

MDS cellular types and subtypes in either cellular classification scheme have different degrees of disordered hematopoiesis, frequencies of transformation to acute leukemia, and prognoses.

Prognostic Scoring Systems

A variety of pathological and risk classification systems have been developed to predict the overall survival of patients with MDS and the evolution from MDS to AML. Major prognostic classification systems include the International Prognostic Scoring System (IPSS), revised as the IPSS-R;[3] the WHO Prognostic Scoring System (WPSS); and the MD Anderson Cancer Center Prognostic Scoring Systems.[4,5] Clinical variables in these systems have included bone marrow and blood myeloblast percentage, specific cytopenias, transfusion requirements, age, performance status, and bone marrow cytogenetic abnormalities.

Supportive Care

The mainstay of treatment for MDS has traditionally been supportive care, particularly for patients with symptomatic cytopenias or who are at high risk of infection or bleeding.[1,2] Transfusions are reserved for the treatment of active bleeding; many centers offer prophylactic platelet transfusions for patients with platelet counts lower than 10,000/mm3. Anemia should be treated with red-cell transfusions to avoid symptoms. For more information, see Fatigue.

No prospective trials have demonstrated the benefit of prophylactic use of myeloid growth factors in asymptomatic neutropenic MDS patients. Similarly, the use of prophylactic antibiotics in such patients is of uncertain benefit. While appropriate use of antibiotics in febrile patients is standard clinical practice, the benefit of myeloid growth factors in such settings is unknown.

The use of erythropoiesis-stimulating agents (ESAs) may improve anemia. The likelihood of response to exogenous erythropoietin administration depends on the pretreatment serum erythropoietin level and baseline transfusion needs.

A meta-analysis summarized the data on erythropoietin in 205 patients with MDS from 17 studies. Responses were most likely in patients who were anemic but who did not yet require a transfusion, patients who did not have ring sideroblasts, and patients who had a serum erythropoietin level lower than 200 IU/L.[3] Effective treatment requires substantially higher doses of erythropoietin than are used for other indications; the minimum effective dose studied is 60,000 IU per week.[4] The use of high-dose darbepoetin (300 µg/dose weekly or 500 µg/dose every 2–3 weeks) has been reported to produce a major erythroid response rate of almost 50% in patients whose endogenous erythropoietin level was lower than 500 mIU/mL.[5,6] Most studies discontinued ESAs in patients who failed to show hematologic improvement after 3 to 4 months of therapy. Average response duration is approximately 2 years.[7]

One decision model found that the likelihood of responding to growth factors was higher in patients with a low serum erythropoietin level (<500 IU/L) and low transfusion needs (<2 units of packed red blood cells every month), but growth factors were rarely effective in patients with a high erythropoietin level and high transfusion needs.[8] Some patients with poor response to erythropoietin alone may have improved response with the addition of low doses of granulocyte colony-stimulating factor (G-CSF) (0.5–1.0 µg/kg/day).[9–11] Rates of response to the combination treatment vary with classification, with responses more likely in patients with refractory anemia and ring sideroblasts (RARS) and less likely in patients with excess blasts.[7] Patients with RARS are unlikely to respond to erythropoietin alone.[3]

The availability of the oral iron-chelating agent deferasirox has led to its widespread use in patients with MDS. While some consensus panels advocate prophylactic iron chelation in patients with ongoing transfusion needs and substantial transfusion history, the impact of iron chelation on survival and disease progression is unknown.[12]

Disease-Modifying Agents

Lower-risk patients (conventionally defined as International Prognostic Scoring System (IPSS) low-risk and intermediate-1–risk groups) who have failed to respond or have ceased responding to ESAs may be treated with one of several disease-modifying agents. The impact of this practice on survival in lower-risk patients is unknown. Whether these drugs should be used following an ESA failure or as up-front therapy has never been determined. In contrast, in higher-risk patients, azacitidine has been shown to improve survival. For more information, see the DNA methyltransferase inhibitors section.

Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

Allogeneic HSCT is the only potentially curative treatment for MDS. Retrospective data suggest cure rates in selected patients ranging from 30% to 60%; outcomes varied with IPSS score at time of transplant, with inferior survival in patients with higher IPSS scores.[32][Level of evidence C3] The role of cytoreductive therapy in reducing the blast percentage before HSCT remains uncertain. Outcomes may not be as good for patients with treatment-related MDS (5-year disease-free survival rate of 8% to 30%).[33]

Although HSCT represents the only treatment modality with curative potential, the relatively high morbidity and mortality of this approach limits its use. A decision analysis predating approval of azacitidine, in patients with a median age younger than 50 years, suggested optimal survival when transplant was delayed until disease progression for lower-risk patients but implemented at diagnosis for higher-risk patients.[34]

Allogeneic stem cell transplant with reduced-intensity conditioning (RIC) has extended transplant as a possible modality for treatment of older patients.[35] In a retrospective analysis of 1,333 patients aged 50 years or older (median, 56 years) who underwent allogeneic transplants for MDS using HLA-matched sibling and unrelated donors, 62% of the patients received RIC HSCT, and the others received standard-dose HSCT. On multivariate analysis, use of RIC and advanced disease stage at transplant were associated with increased relapse (hazard ratio [HR] of 1.44 and 1.51, respectively).[35][Level of evidence C3] The predictors of non-relapse mortality included advanced disease stage (HR, 1.43), use of an unrelated donor, and standard-dose HSCT (HR, 1.27). The 4-year OS rate was similar in both groups (30% after myeloablative conditioning vs. 32% in RIC.[35]

Therapy-Related Myeloid Neoplasms

In the absence of prospective data, therapy-related myeloid neoplasms are treated similarly to de novo MDS.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of myelodysplastic syndromes. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewer for Myelodysplastic Syndromes Treatment is:

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic Syndromes Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/myeloproliferative/hp/myelodysplastic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389450]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s Email Us.

Myeloproliferative neoplasms are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells, or platelets.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time.

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A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

Enlarge

In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases.

The following are types of myeloproliferative neoplasms.

The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Myeloproliferative neoplasms include:

These types are described below. Myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made.

Tests that examine the blood and bone marrow are used to diagnose myeloproliferative neoplasms.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

Polycythemia vera is a disease in which too many red blood cells are made in the bone marrow.

In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis.

Symptoms of polycythemia vera include headaches and a feeling of fullness below the ribs on the left side.

Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have:

Special blood tests are used to diagnose polycythemia vera.

In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells.

Essential thrombocythemia is a disease in which too many platelets are made in the bone marrow.

Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow.

Patients with essential thrombocythemia may have no signs or symptoms.

Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have:

Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke and heart attack, or pulmonary embolism and deep vein thrombosis in people older than 60 years, who have had blood clots or high white blood cell counts in the past. In some people, essential thrombocythemia may become acute leukemia.

Certain factors affect prognosis (chance of recovery) and treatment options for essential thrombocythemia.

Prognosis and treatment options depend on:

Primary myelofibrosis is a disease in which abnormal blood cells and fibers build up inside the bone marrow.

The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells.

Symptoms of primary myelofibrosis include pain below the ribs on the left side and feeling very tired.

Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have:

Certain factors affect prognosis (chance of recovery) and treatment options for primary myelofibrosis.

The prognosis depends on:

Chronic eosinophilic leukemia is a disease in which too many white blood cells (eosinophils) are made in the bone marrow.

Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia.

Signs and symptoms of chronic eosinophilic leukemia include fever and feeling very tired.

Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have:

There is no standard staging system for myeloproliferative neoplasms.

The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment.

There are different types of treatment for patients with myeloproliferative neoplasms.

Different types of treatments are available for patients with myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The following types of treatment are used:

High-dose chemotherapy with stem cell transplant

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.

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New types of treatment are being tested in clinical trials.

For some people, joining a clinical trial may be an option. There are different types of clinical trials for people with cancer. For example, a treatment trial tests new treatments or new ways of using current treatments. Supportive care and palliative care trials look at ways to improve quality of life, especially for those who have side effects from cancer and its treatment.

You can use the clinical trial search to find NCI-supported cancer clinical trials accepting participants. The search allows you to filter trials based on the type of cancer, your age, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more about clinical trials, including how to find and join one, at Clinical Trials Information for Patients and Caregivers.

Treatment for myeloproliferative neoplasms may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Follow-up care may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute’s (NCI’s) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

Reviewers and Updates

Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary (“Updated”) is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become “standard.” Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI’s website. For more information, call the Cancer Information Service (CIS), NCI’s contact center, at 1-800-4-CANCER (1-800-422-6237).

Permission to Use This Summary

PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].”

The best way to cite this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.

Disclaimer

The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us.

Chronic myeloid leukemia is a disease in which the bone marrow makes too many white blood cells.

Chronic myeloid leukemia (also called CML or chronic myelogenous leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age and rarely occurs in children.

Enlarge

Leukemia may affect red blood cells, white blood cells, and platelets.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell.

A myeloid stem cell becomes one of three types of mature blood cells:

Enlarge

In CML, too many myeloblasts (a type of immature white blood cell) form in the blood and bone marrow, and the disease worsens as the number of myeloblasts increases.

CML is one of a group of diseases called myeloproliferative neoplasms.

Signs and symptoms of chronic myeloid leukemia include weight loss and tiredness.

These and other signs and symptoms may be caused by CML or by other conditions. Check with your doctor if you have:

Sometimes CML does not cause any symptoms at all.

Most people with chronic myeloid leukemia have a gene mutation (change) called the Philadelphia (Ph) chromosome.

Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “Philadelphia chromosome.” It results in the bone marrow making a protein, called tyrosine kinase, that causes too many stem cells to become white blood cells (granulocytes or blasts).

The Philadelphia chromosome is not passed from parent to child.

Enlarge

Tests that examine the blood and bone marrow are used to diagnose chronic myeloid leukemia.

In addition to asking about your personal and family health history and doing a physical exam to check for signs of disease, such as an enlarged spleen, your doctor may perform the following tests and procedures:

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis and treatment options depend on:

After chronic myeloid leukemia has been diagnosed, tests are done to find out if the cancer has spread.

The extent or spread of cancer is usually described as stages. In chronic myeloid leukemia (CML), the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The information from tests and procedures done to diagnose chronic myeloid leukemia is also used to plan treatment.

Chronic myeloid leukemia has 3 phases.

As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of signs or symptoms determine the phase of the disease.

Chronic myeloid leukemia can relapse (return) after it has been treated.

In relapsed CML, the number of blast cells increases after a remission.

There are different types of treatment for patients with chronic myeloid leukemia.

Different types of treatments are available for chronic myeloid leukemia (CML). You and your cancer care team will work together to decide your treatment plan, which may include more than one type of treatment. Many factors will be considered, such as the stage of the cancer, your overall health, and your preferences. Your plan will include information about your cancer, the goals of treatment, your treatment options and the possible side effects, and the expected length of treatment.

Talking with your cancer care team before treatment begins about what to expect will be helpful. You’ll want to learn what you need to do before treatment begins, how you’ll feel while going through it, and what kind of help you will need. To learn more, visit Questions to Ask Your Doctor About Treatment.

The following types of treatment are used:

High-dose chemotherapy with stem cell transplant (SCT)

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.

Learn more about Stem Cell Transplants in Cancer Treatment.

Enlarge

New types of treatment are being tested in clinical trials.

For some people, joining a clinical trial may be an option. There are different types of clinical trials for people with cancer. For example, a treatment trial tests new treatments or new ways of using current treatments. Supportive care and palliative care trials look at ways to improve quality of life, especially for those who have side effects from cancer and its treatment.

You can use the clinical trial search to find NCI-supported cancer clinical trials accepting participants. The search allows you to filter trials based on the type of cancer, your age, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more about clinical trials, including how to find and join one, at Clinical Trials Information for Patients and Caregivers.

Treatment for chronic myeloid leukemia may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Follow-up care may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute’s (NCI’s) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of chronic myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

Reviewers and Updates

Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary (“Updated”) is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become “standard.” Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI’s website. For more information, call the Cancer Information Service (CIS), NCI’s contact center, at 1-800-4-CANCER (1-800-422-6237).

Permission to Use This Summary

PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].”

The best way to cite this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/leukemia/patient/cml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389183]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.

Disclaimer

The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us.