Treatment Options for Myelofibrosis

Treatment Options

Watch and Wait
People with no symptoms are generally not treated at the time of diagnosis. Some people remain stable and symptom-free for several years. It is very important for anyone who has been diagnosed with myelofibrosis to be closely monitored through regular medical check-ups to look for any signs or symptoms of disease progression.

Drug Therapies
Many kinds of drugs are used to treat myelofibrosis. They work in different ways and have different side effects. Ask your doctor or nurse to tell you about possible side effects before starting a new drug.

Cytoreductive Therapies (e.g. hydroxyurea)
These drugs reduce the blood counts and are usually given by mouth (orally). The main side effects is lowering of the blood counts (in some cases too much).

JAK Inhibitors
These new kinds of drugs work by targeting the JAK1 and/or JAK2 proteins that help regulate blood. They can treat and lessen some of the symptoms of myelofibrosis, including an enlarged spleen, night sweats, itching and bone or muscle pain. They can work in both patients that have a JAK2 mutation and those that do not. Two JAK inhibitors, ruxolitinib (Jakafi®) and fedratinib (Inrebic®), are approved to treat myelofibrosis.

Immunomodulators (IMiDs)
These drugs (e.g. thalidomide, lenalomide) fight cancer cells by helping the immune system work normally. They sometimes are used to treat symptoms such as anemia, an enlarged spleen, bone pain and high platelet count.

Androgen Therapy
This male hormone treatment can increase red blood cell production. These drugs may help with the symptoms of severe anemia.

Recombinant Erythropoietin
This treatment helps regulate red blood cell production. An injection is given into a muscle or under the skin.

Anagrelide Hydrochloride
This drug, given orally, may be used in MF patients who have an extremely high platelet count.

Radiation Therapy
Radiation uses high-powered beams, such as X-rays, to kill cells. Radiation therapy can help reduce the size of the spleen, when surgical removal isn’t an option. It is also used to treat bone pain and tumors outside of the bone marrow.

Surgical Removal of the Spleen (Splenectomy)
If your spleen is enlarged and causing harmful complications and pain, and other interventions have not been successful, you may benefit from having your spleen surgically removed. Your doctor will consider the risks and benefits of your particular situation and determine if this is a feasible option. If surgery is done, you should be closely monitored for complications.

Stem Cell Transplantation
This is the only type of treatment that has the potential to cure myelofibrosis. It is a very high-risk treatment that depends on the availability of a suitable donor. It is not an option for everyone.

Allogeneic Stem Cell Transplant
In this type of transplant, a person with myelofibrosis receives high doses of chemotherapy or radiation to destroy the diseased bone marrow. The donor stem cells (from someone other than the patient) are then infused into the patient.

Non-myeloblative Stem Cell Transplant
This type of transplant uses lower doses of chemotherapy or radiation prior to the infusion of donor cells. This type of transplant can be used in older patients, who may not be eligible for an allogeneic transplant.

Clinical Trials
Other treatments may be available through clinical trials. Clinical trials can offer the opportunity to benefit from the latest treatments or combinations of treatments while helping advance knowledge of the disease and its treatment options. Ask your health care team if a clinical trial might be right for you.