General Information About Myelodysplastic/ Myeloproliferative Neoplasms

KEY POINTS

• Myelodysplastic/myeloproliferative neoplasms are a group of diseases in which the bone marrow makes too many white blood cells.
• Myelodysplastic/myeloproliferative neoplasms have features of both myelodysplastic syndromes and myeloproliferative neoplasms.
• There are different types of myelodysplastic/myeloproliferative neoplasms.
• Tests that examine the blood and bone marrow are used to diagnose myelodysplastic/myeloproliferative neoplasms.

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.

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:

• Red blood cells that carry oxygen and other substances to all tissues of the body.
• Granulocytes, which are white blood cells that help fight infection and disease.
• Platelets that form blood clots to stop bleeding.

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.

There are different types of myelodysplastic/myeloproliferative neoplasms.

The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following:

• Chronic myelomonocytic leukemia (CMML).
• Juvenile myelomonocytic leukemia (JMML).
• Atypical chronic myelogenous leukemia (CML).

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.

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.

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

Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia.

In JMML, 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.

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

In atypical chronic myelogenous 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.

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

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

The following tests and procedures may be used:

• Physical exam and health history: An exam of the body to check general signs of health, including checking for signs of disease such as an enlarged spleen and liver. A history of the patient’s health habits and past illnesses and treatments will also be taken.
• Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following:
  • The number of red blood cells and platelets.
  • The number and type of white blood cells.
  • The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
  • The portion of the sample made up of red blood cells.

• Peripheral blood smear: A procedure in which a sample of blood is checked for blast cells, the number and kinds of white blood cells, the number of platelets, and changes in the shape of blood cells.
• Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease.
• Bone marrow aspiration and biopsy: The removal of a small piece of bone and bone marrow by inserting a needle into the hipbone or breastbone. A pathologist views both the bone and bone marrow samples under a microscope to look for abnormal cells.

  The following tests may be done on the sample of tissue that is removed:

  • Cytogenetic analysis: A laboratory test in which the chromosomes of cells in a sample of bone marrow or blood are counted and checked for any changes, such as broken, missing, rearranged, or extra chromosomes. Changes in certain chromosomes may be a sign of cancer. Cytogenetic analysis is used to help diagnose cancer, plan treatment, or find out how well treatment is working. The cancer cells in myelodysplastic/myeloproliferative neoplasms do not contain the Philadelphia chromosome that is present in chronic myelogenous leukemia.
  • Immunocytochemistry: A laboratory test that uses antibodies to check for certain antigens (markers) in a sample of a patient’s bone marrow. The antibodies are usually linked to an enzyme or a fluorescent dye. After the antibodies bind to the antigen in the sample of the patient's bone marrow, the enzyme or dye is activated, and the antigen can then be seen under a microscope. This type of test is used to help diagnose cancer and to tell the difference between myelodysplastic/myeloproliferative neoplasms, leukemia, and other conditions.

KEY POINTS

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

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.

KEY POINTS

• There are different types of treatment for patients with myelodysplastic/myeloproliferative neoplasms.
• Five types of standard treatment are used:
  • Chemotherapy
  • Other drug therapy
  • Stem cell transplant
  • Supportive care
  • Targeted therapy
• New types of treatment are being tested in clinical trials.
• Treatment for myelodysplastic/myeloproliferative neoplasms may cause side effects.
• Patients may want to think about taking part in a clinical trial.
• Patients can enter clinical trials before, during, or after starting their cancer treatment.
• Follow-up tests may be needed.

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.

Five types of standard treatment are used:

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug.

Other drug therapy

13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act.

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.

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do.

• Tyrosine kinase inhibitor (TKI) therapy: TKI therapy blocks signals that tumors need to grow. TKIs block the enzyme, tyrosine kinase, that causes stem cells to become more blood cells (blasts) than the body needs. Imatinib mesylate (Gleevec) is used to treat myelodysplastic/myeloproliferative neoplasm, unclassifiable. Other targeted therapy drugs are being studied in the treatment of juvenile myelomonocytic leukemia (JMML).

Treatment for myelodysplastic/myeloproliferative neoplasms may cause side effects.

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.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order 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). These tests are sometimes called follow-up tests or check-ups.

Treatment of Chronic Myelomonocytic Leukemia

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of chronic myelomonocytic leukemia (CMML) may include the following:

• Chemotherapy with one or more agents.
• Stem cell transplant.
• A clinical trial of a new treatment.

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done.

Treatment of Juvenile Myelomonocytic Leukemia

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of juvenile myelomonocytic leukemia (JMML) may include the following:

• Combination chemotherapy.
• Stem cell transplant.
• 13-cis-retinoic acid therapy.
• A clinical trial of a new treatment, such as targeted therapy.

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done.

Treatment of Aypical Chronic Myelogenous Leukemia

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of atypical chronic myelogenous leukemia (CML) may include the following:

• Chemotherapy.

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done.

Treatment of Myelodysplastic/ Myeloproliferative Neoplasm, Unclassifiable

For information about the treatments listed below, see the Treatment Option Overview section.

Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following:

• Supportive care treatments to manage problems caused by the disease such as infection, bleeding, and anemia.
• Targeted therapy (imatinib mesylate).

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done.

To Learn More About Myelodysplastic/ Myeloproliferative Neoplasms

For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following:

• Myeloproliferative Neoplasms Home Page
• Stem Cell Transplants in Cancer Treatment
• Drugs Approved for Myeloproliferative Neoplasms
• Targeted Therapy to Treat Cancer

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

• About Cancer
• Staging
• Chemotherapy and You: Support for People With Cancer
• Radiation Therapy and You: Support for People With Cancer
• Coping with Cancer
• Questions to Ask Your Doctor about Cancer
• For Survivors and Caregivers

This page lists cancer drugs approved by the Food and Drug Administration (FDA) for myeloproliferative neoplasms or myelodysplastic syndromes. The list includes generic and brand names. This page also lists common drug combinations used in myeloproliferative neoplasms or myelodysplastic syndromes. The individual drugs in the combinations are FDA-approved. However, drug combinations themselves usually are not approved, but are widely used.

There may be drugs used in myeloproliferative neoplasms or myelodysplastic syndromes that are not listed here.

Drugs Approved for Myeloproliferative Neoplasms or Myelodysplastic Syndromes

• Adriamycin PFS (Doxorubicin Hydrochloride)
• Adriamycin RDF (Doxorubicin Hydrochloride)
• Arsenic Trioxide
• Azacitidine
• Besremi (Ropeginterferon Alfa-2b-njft)
• Cerubidine (Daunorubicin Hydrochloride)
• Clafen (Cyclophosphamide)
• Cyclophosphamide
• Cytarabine
• Cytosar-U (Cytarabine)
• Cytoxan (Cyclophosphamide)
• Dacogen (Decitabine)
• Dasatinib
• Daunorubicin Hydrochloride
• Decitabine
• Decitabine and Cedazuridine
• Doxorubicin Hydrochloride
• Fedratinib Hydrochloride
• Gleevec (Imatinib Mesylate)
• Imatinib Mesylate
• Imetelstat Sodium
• Inqovi (Decitabine and Cedazuridine)
• Inrebic (Fedratinib Hydrochloride)
• Ivosidenib
• Jakafi (Ruxolitinib Phosphate)
• Nilotinib
• Pacritinib Citrate
• Pemazyre (Pemigatinib)
• Pemigatinib
• Ropeginteferon Alfa-2b-njft
• Rubidomycin (Daunorubicin Hydrochloride)
• Ruxolitinib Phosphate
• Rytelo (Imetelstat Sodium)
• Sprycel (Dasatinib)
• Tarabine PFS (Cytarabine)
• Tasigna (Nilotinib)
• Tibsovo (Ivosidenib)
• Trisenox (Arsenic Trioxide)
• Vidaza (Azacitidine)
• Vonjo (Pacritinib Citrate)

Drug Combinations Used in Myeloproliferative Neoplasms or Myelodysplastic Syndromes

• ADE