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 characterized by which type of blood cell is in abundance, including:

  • Polycythemia vera, which is characterized by too many red blood cells
  • Myelofibrosis, which occurs when the bone marrow produces excess collagen or fibrous tissue
  • Essential thrombocythemia, which is characterized by too many platelets1

Chronic myeloid leukemia (CML) is also classified as an MPN, as CML can result in an abundance of white blood cells.1,2

Treatment for MPN is determined on several factors, including the type of MPN, whether the individual is experiencing symptoms from their condition, and the age and overall health of the individual.

Types of treatment for myeloproliferative neoplasms

There are several different types of treatment that may be used for MPN, including:

  • Watchful waiting
  • Phlebotomy
  • Platelet apheresis
  • Blood transfusions
  • Chemotherapy
  • Other medications
  • Radiation therapy
  • Surgery
  • Immunotherapy
  • Targeted therapy
  • Stem cell transplant1

Watchful waiting

Watchful waiting may be recommended if a person with MPN is not experiencing symptoms from their disease. During watchful waiting, the person does not receive treatment for the blood cancer, although problems like infections are treated. A person’s health is monitored closely during watchful waiting, and doctors will watch for any changes in their condition and the potential appearance of, or change in, symptoms.1


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 may be used in the treatment of certain cases of MPN. Chemotherapy medications may be used in combination, and chemotherapy drugs 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, and it may be directed at the spleen in certain cases of MPN.1

Other medications

In addition to the chemotherapy drugs, other drugs may be used to treat MPN, including prednisone or danazol for anemia in people with primary myelofibrosis, or anagrelide therapy or low dose aspirin may be utilized in people with essential thrombocytopenia. Patients should take their medication as prescribed by their healthcare provider. Patients should talk to their healthcare provider if they have any questions, or if they have questions regarding their medication regimen.1


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

Targeted therapy

Targeted therapies may be used in the treatment of 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 by focusing on particular characteristics that are unique to certain MPN cells. While chemotherapy drugs are often cytotoxic, meaning they kill specific cells, targeted therapy is typically cytostatic, meaning it blocks the growth of certain cells. One type of targeted therapy that may be used to treat MPN is tyrosine kinase inhibitors. Tyrosine kinase inhibitors block specific proteins, and this action can keep MPN cells from growing and may kill them.2

Stem cell transplants

Stem cell transplants are used in combination with high doses of chemotherapy and may be a treatment option for certain people with MPN. The high dose of chemotherapy destroys the MPN cells and also damages healthy blood cells. The transplant of stem cells, immature cells that can become new blood cells, is given to restore the bone marrow. The stem cells may be gathered from the patient prior to chemotherapy (called an autologous transplant), or they may be given by a donor (called an allogeneic transplant). Not everyone is a candidate for stem cell transplants, however, as the high doses of chemotherapy can be very taxing on a person’s body and may not be tolerated by older patients 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, and they offer patients a chance to receive the latest treatments and be closely monitored by healthcare professionals. Clinical trials can be found by talking to a healthcare provider or through the website Patients can discuss treatment options with their doctor to determine if they might be eligible to participate in a clinical trial.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: October 2020