Treatment Options for Myelofibrosis

Medically reviewed by Graham Rogers, MD on August 1, 2016Written by Faith Black on August 1, 2016

Myelofibrosis (MF) is a rare, but serious bone marrow disorder. Currently, there is no reliable cure for MF. Treatments focus more on the symptoms and complications people with MF experience.

Because of this, many people with MF who don’t have symptoms can go without treatment for years. Treatment options for MF include:

  • drug therapies
  • blood transfusions
  • radiation therapy
  • splenectomy
  • stem cell transplant
  • clinical trials

Drug therapies

JAK inhibitors

The newest type of drug used to fight MF is a class of drugs called Janus-associated kinase (JAK) inhibitors. This type of drug targets the JAK enzymes. These enzymes are involved in the production of red blood cells. By inhibiting or stopping these enzymes, JAK inhibitors can slow the production of blood cells.

Ruxolitinib (Jakafi) is the only drug of this class approved by the U.S. Food and Drug Administration (FDA). In some patients, this drug has decreased spleen size and reduced other symptoms of MF.

Chemotherapy

Chemotherapy is another drug therapy used to treat MF. It targets cells that are growing and dividing rapidly, like cancer cells or the mutated blood stem cells in MF. However, chemotherapy can’t separate cancer cells from other healthy cells that grow and divide quickly. This includes hair cells and cells that line the GI tract. Because of this, chemotherapy can cause many side effects, such as hair loss, nausea, vomiting, diarrhea, and bleeding.

Androgen therapy

Androgen therapy is another type of medication used to treat the low blood count in people with MF. This type of drug is a lab-made version of male hormones. It’s used to increase red blood cell production. About one in three people with MF will see an improvement in their anemia with this treatment. However, androgens can be toxic to the liver. People on this therapy require increased monitoring to track liver functions. This includes regular blood tests and ultrasounds. Androgen therapy may also cause masculinizing effects, such as facial hair growth in women.

Corticosteroids

Corticosteroids have been shown to improve anemia in about one in three people with MF. Steroid compounds are used to treat many different diseases, and some promising results have been seen for MF patients with significant anemia.

Blood transfusions

In patients with severe anemia, drug therapy may not be enough to keep the red blood cell count high enough. Regular blood transfusions can help increase blood cell count and reduce symptoms of anemia such as bleeding, bruising, weakness, and fatigue.

Radiation therapy

Radiation therapy is used to kill cells through strong X-rays or other types of radiation. It’s a common treatment in cancer, as it can stop tumor cells from growing. In MF, radiation is used in a small group of patients to shrink the size of the spleen, relive bone pain, and shrink tumors growing outside of the bone marrow.

Splenectomy

A splenectomy is an option for people whose spleens have become very large, are causing them pain or other complications, and who haven’t responded to other treatments. During this procedure, a surgeon will remove the spleen.

Before deciding to remove the spleen, you should talk with your doctor about the benefits and risks of the procedure. Complications from a splenectomy can include:

  • blood clot formation, leading to a stroke or heart attack
  • pulmonary embolism
  • venous thrombosis
  • liver enlargement
  • infections
  • an increase in platelet count

Stem cell transplant

An allogenic stem cell transplant, also called a bone marrow transplant, is the only treatment with the possibility to cure MF. Before the transplant procedure, high doses of radiation or chemotherapy are used to destroy the diseased bone marrow. After the diseased bone marrow has been killed, you receive healthy blood-forming bone marrow stem cells from a compatible donor. The transplanted, healthy stem cells spread to your bone marrow and replace the diseased marrow. Then they start producing healthy red and white blood cells, as well as platelets.

This procedure is very high risk. Due to the age and health of many people with MF, very few people are eligible for this procedure. Researchers are trying to find new types of stem cell transplants that would involve less chemotherapy or radiation, or that have lower chances of life-threatening side effects.

Clinical trials

Clinical trials are available for people with MF who are interested in trying therapies or treatments that aren’t available to the general public yet. Clinical trials are studies done to see how well a new treatment or procedure works, before making it available to everyone. These trials are important to medicine and they give treatment options to people who may not have many other choices.

Some of the current clinical trials for MF include other drug therapies focusing on inhibiting the JAK enzymes, as well as drugs that inhibit other enzymes known to be at work in cancer. Researchers are also investigating combinations of different drugs as well as different ways to perform a stem cell transplant.

Making the decision to participate in a clinical trial can be scary, but it can open up many treatment doors that are otherwise closed. If you’re interested in being a part of a clinical trial, talk with your doctor to find the best one for you.

While there’s no reliable cure for MF, there’s no reason to give up hope. Treatments are aimed at easing your symptoms and improving your quality of life. Being open and honest with your healthcare team can ensure that you get on a treatment plan that’s best for your needs.

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