Myeloproliferative Neoplasm (MPN) Treatment

Reviewed by: HU Medical Review Board | Last reviewed: May 2023

Myeloproliferative neoplasms (MPN) are a group of blood diseases. They develop from overactive blood cells in the bone marrow. The different forms of MPN are defined by which type of blood cell is overproduced. These forms include:1

  • Polycythemia vera – too many red blood cells
  • Myelofibrosis – too much collagen or fibrous tissue
  • Essential thrombocythemia – 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

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

Types of treatment for myeloproliferative neoplasms

Many types of treatment may be used for MPN, including:1-3

  • Watchful waiting
  • Phlebotomy
  • Platelet apheresis
  • Blood transfusions
  • Chemotherapy
  • Other medicines such as aspirin, anemia treatments, or erythropoietic growth drugs
  • Radiation therapy
  • Surgery
  • Immunotherapy
  • Targeted therapy, such as tyrosine kinase inhibitors
  • Stem cell transplant

Watchful waiting

Your doctor may recommend watchful waiting if you do not have symptoms from your MPN. During watchful waiting, you do not receive treatment for the blood cancer. But problems like infections are treated. Your health is closely monitored. Doctors will watch for any changes in your condition and symptoms.1

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Phlebotomy is a procedure where blood is removed from the body. Phlebotomy can also be used to treat certain types of MPN by removing excess red blood cells from the body.1

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 an MPN. The transfusion replaces blood cells that have been depleted by treatment or by the disease itself.1


Chemotherapy may be used to treat certain cases of MPN. The chemotherapy drugs may be taken by mouth (orally) or injected into a vein or muscle.1

Chemotherapy works by targeting fast-growing cells, such as MPN cells. But there are other fast-growing cells in the body that chemotherapy can also affect. These include cells in the digestive tract and hair.1

Other medicines

Other drugs may be used to treat MPN, including:1-3

  • Prednisone or danazol for anemia in people with primary myelofibrosis
  • Luspatercept, lenalidomide, immunosuppressive therapy, or erythropoietic growth drugs to treat anemia in some people with MPN
  • Anagrelide therapy or low-dose aspirin in people with essential thrombocythemia or polycythemia vera


In some cases, a person with MPN may have surgery to remove an enlarged spleen. This surgery is called a splenectomy. This surgery does not cure MPN, but it can relieve some of the symptoms.1

Radiation therapy

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


Immunotherapy 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 can help block or slow the growth and spread of certain MPN cells. In contrast, chemotherapy drugs are designed to kill specific cells.1,2,4

Tyrosine kinase inhibitors are one type of targeted therapy used to treat MPN. These inhibitors block specific proteins. Blocking these proteins can keep MPN cells from growing and may kill the MPN cells. Ruxolitinib is a type of tyrosine kinase inhibitor called a janus kinase inhibitor. Ruxolitinib can treat some types of MPN.1,2,4

Stem cell transplants

Stem cells are immature cells that can become new blood cells. Stem cell transplants are used along with high doses of chemotherapy. The high dose of chemotherapy destroys the MPN cells. But it also damages healthy blood cells.1

A stem cell transplant is given to restore bone marrow. The stem cells may be gathered from the person before chemotherapy or given by a donor.1

But high doses of chemotherapy can be very hard on a person’s body. These high doses may not be tolerated by older adults or those with other health problems. So not everyone is a candidate for stem cell transplants.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 study the safety and effectiveness of new treatments. They also offer people a chance to receive new treatments and be closely monitored by doctors.1,5

You can talk to your doctor or visit the website to learn more about clinical trials. Your doctor can help you decide if a clinical trial may be right for you.1,5

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.