Myeloproliferative Neoplasm (MPN) Treatment

Myeloproliferative neoplasms (MPN) are a group of blood cancers that develop from immature blood cells in the bone marrow. The different forms of MPN are defined by which type of blood cell is in abundance, including:1

  • Polycythemia vera, which is defined by too many red blood cells
  • Myelofibrosis, which occurs when the bone marrow produces too much collagen or fibrous tissue
  • Essential thrombocythemia, which is defined by too many platelets

Chronic myeloid leukemia (CML) is also classified as an MPN. This is because CML can result in too many white blood cells. Treatment for MPN is based on several factors, including:1,2

  • The type of MPN
  • Whether the person is experiencing symptoms from their condition
  • The age and overall health of the person

Types of treatment for myeloproliferative neoplasms

Many types of treatment may be used for MPN, including:1

  • Watchful waiting
  • Phlebotomy
  • Platelet apheresis
  • Blood transfusions
  • Chemotherapy
  • Other medicines
  • Radiation therapy
  • Surgery
  • Immunotherapy
  • Targeted therapy
  • Stem cell transplant

Watchful waiting

Watchful waiting may be recommended if you do not have symptoms from MPN. During watchful waiting, you do not receive treatment for the blood cancer. However, problems like infections are treated. Your health is closely monitored during watchful waiting. Doctors will watch for any changes in your condition and the potential appearance of, or change in, symptoms.1

Phlebotomy

Phlebotomy is a procedure where blood is removed from the body, such as during routine testing. Phlebotomy can also be used as a treatment for certain types of MPN, such as to remove excess red blood cells from the body.1,2

Platelet apheresis

Platelet apheresis is a procedure where platelets are removed from the blood with the use of a special machine.1

Blood transfusions

Blood transfusions may be given to people with MPN to replace blood cells that have been depleted by treatment or by the disease itself.1

Chemotherapy

Chemotherapy may be used in the treatment of certain cases of MPN. Chemotherapy drugs may be used together, and they may be taken by mouth or injected into a vein or muscle. Chemotherapy works by targeting fast-growing cells, such as MPN cells. However, there are other fast-growing cells in the body that can also be affected, such as those in the gastrointestinal tract and hair.1

Radiation therapy

Radiation therapy uses high-energy radiation. It may be directed at the spleen in certain cases of MPN.1

Other medicines

Along with chemotherapy drugs, other drugs may be used to treat MPN, including:1

  • Prednisone or danazol for anemia in people with primary myelofibrosis
  • Anagrelide therapy or low-dose aspirin in people with essential thrombocytopenia

Surgery

In some cases, a person with MPN may have surgery to remove an enlarged spleen. This surgery is called a splenectomy. While this surgery does not cure MPN, it can relieve some of the symptoms, such as when an enlarged spleen presses on other organs like the stomach.1

Immunotherapy

Immunotherapy, or biologic therapy, is a type of treatment that boosts the body’s immune system to fight the MPN cells. Immunotherapies called interferon alpha and pegylated interferon alpha may be used to treat certain cases of MPN.1

Targeted therapy

Targeted therapies may be used to treat certain cases of MPN. Targeted therapies can help block or slow the spread of certain MPN cells by interfering with specific areas of cells that are involved in the MPN cell’s growth. Or, they focus on features that are unique to certain MPN cells.2

Chemotherapy drugs are often cytotoxic, meaning they kill specific cells. However, targeted therapy is typically cytostatic, meaning it blocks the growth of certain cells. Tyrosine kinase inhibitors are 1 type of targeted therapy used to treat MPN. They block specific proteins, which can keep MPN cells from growing and may kill them.2

Stem cell transplants

Stem cell transplants are used along with high doses of chemotherapy. They may be a treatment option for some people with MPN. The high dose of chemotherapy destroys the MPN cells, but it also damages healthy blood cells.1

Stem cells are immature cells that can become new blood cells. A stem cell transplant is given to restore bone marrow. The stem cells may be gathered from the person before chemotherapy. This is called an autologous transplant. Or, they may be given by a donor. This is called an allogeneic transplant.1

Not everyone is a candidate for stem cell transplants. However, high doses of chemotherapy can be very hard on a person’s body and may not be tolerated by older adults or those with other health problems.1

Clinical trials

Clinical trials are a type of research where new treatments are studied. Clinical trials are an important part of the scientific process to find and prove the safety and effectiveness of new treatments. They offer people a chance to receive the latest treatments and be closely monitored by doctors. You can learn more about clinical trials by talking to your doctor or visiting the ClinicalTrials.gov website. Your doctor can help you decide if a clinical trial may be right for you.1,3

Editor's note: The classification of MPN has evolved over time. Some organizations may classify MPN as either a blood cancer or a blood disorder.

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Written by: Emily Downward | Last reviewed: April 2021