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Chemotherapy and Drug Therapy

Drug therapy is used for myelofibrosis (MF) to lower platelet counts. Your doctor may use one or more of the following treatments:

Janus-associated kinase (JAK) inhibitor, such as ruxolitinib (JakafiTM). This drug class inhibits enzymes called JAK1 and JAK2, which are involved in the production of blood cells. Jakafi, given by mouth, is the first JAK inhibitor and currently the only drug approved by the FDA to treat symptoms and signs of MF, including an enlarged spleen, night sweats, itching and bone or muscle pain. It is indicated for treatment of patients with intermediate- or high-risk myelofibrosis, including primary myelofibrosis, post polycythemia vera myelofibrosis and post essential thrombocythemia myelofibrosis.

Patients should be aware that after discontinuation of Jakafi, myelofibrosis signs and symptoms are expected to return. There have been isolated cases of patients discontinuing Jakafi during acute intervening illnesses after which the patient?s clinical course continued to worsen. It has not been established whether discontinuation of therapy contributed to the clinical course of these patients. When discontinuing Jakafi therapy for reasons other than thrombocytopenia, gradual tapering of the dose of Jakafi may be considered.
For more details, please see the full prescribing information for Jakafi available at www.accessdata.fda.gov/drugsatfda_docs/label/2011/202192lbl.pdf or contact an LLS Information Specialist.

Hydroxyurea (Hydrea®). Hydroxyurea is a commonly used chemotherapy drug, given by mouth. It's used to:

  • decrease very high platelet counts
  • decrease size and associated complications of an enlarged spleen
  • decrease night sweats and weight loss
  • decrease white blood cell counts.

Interferon alfa (Intron® A, Roferan-A®). Interferon alfa is a synthetic version of a substance made by cells to fight infection and tumors. It is given by intramuscular or subcutaneous injection and has been used to treat an enlarged spleen, bone pain and high platelet counts in selected MF patients.

Androgens, such as oxymetholone (Anadrol®) and danazol (Danocrine®). Androgens are drugs that are synthetic versions (analogues) of male hormones and are given by mouth. These agents can promote red cell production and relieve severe anemia symptoms. Androgen treatment helps improve anemia in one in three MF patients. Because of androgen's toxic effects on the liver, patients must undergo blood tests and ultrasound imaging to track liver function. Androgens can cause women to develop masculine characteristics such as facial hair.

Erythropoietin (Epogen(R), Procrit(R)). This treatment is given intravenously (IV) or by subcutaneous injection and helps regulate red cell production. A small number of patients have been treated successfully with the hormone erythropoietin. However, it generally hasn't been a successful treatment for MF-related anemia.

Glucocorticoids, such as prednisone. These drugs, given by mouth, are steroid compounds used to treat many conditions. They may benefit patients who have significant anemia. When treated with prednisone, anemia improves in about one in three patients.

Bisphosphonates, such as zoledronic acid (Zometa®). Bisphosphonates may help relieve bone pain and improve blood counts. These drugs are given intravenously (IV).

Anagrelide (Agrylin®). Anagrelide is a noncytotoxic drug (an agent that doesn't kill cells), given by mouth, may be used to treat patients who have a very high platelet count, especially following removal of the spleen (splenectomy).

If you would like to read about these drugs individually, including information about side effects, click here.

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last updated on Wednesday, April 24, 2013

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