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Myelofibrosis

About Myelofibrosis (MF)

Myelofibrosis (MF) develops when uncontrolled signaling in the JAK pathway – which regulates blood cell production – causes faulty blood cell production, resulting in an enlarged spleen.1,2,3 Patients may also suffer debilitating symptoms including fever, moderate fatigue, intractable pruritus (itchiness), night sweats, weight loss, bone or muscle pain, poor quality of life and shortened survival.4

MF is a type of Philadelphia (Ph) chromosome-negative “myeloproliferative neoplasm,” [whereas Ph chromosome-positive is chronic myelogenous leukemia (CML)], a group of diseases in which specific types of blood cells are overproduced in the body and disrupt their normal functioning. Other Philadelphia chromosome-negative myeloproliferative neoplasms include essential thrombocythemia (ET) and polycythemia vera (PV). About 10% to 15% of MF cases begin with ET or PV and are called “secondary myelofibrosis;” the rest of cases develop on their own and are referred to as “primary myelofibrosis”.1,5

Treatment of Myelofibrosis

  • For most people, MF treatment does not cure the disease. Rather, the goal of treatment is to reduce symptoms and improve quality of life. This means that the treatment patients receive for MF will depend on the symptoms. It is, therefore, important that patients and caregivers understand the symptoms to collaborate with a hematologist to create an optimal treatment plan.
  • Remember, even mild symptoms can have a significant impact on day-to-day life and if they do, it’s important to address these with a hematologist.

Questions to ask your doctor

  • How can I assess my symptoms?
  • How will my MF related symptoms impact my quality of life?
  • What are my treatment choices, specifically in terms of medication therapy, splenectomy and transplant?
  • What are the expected benefits of each kind of treatment?
  • What are the risks of each treatment?
  • What are the side effects of each treatment?
  • Is there a benefit to starting treatment prior to the development of debilitating symptoms?
  • How will we know if the treatment is working?
  • How will each treatment affect my daily life?
  • Are there new treatments or clinical trials I should consider?

SpotlightonMPN.com website

SpotlightonMPN.com to learn more about myelofibrosis and other myeloproliferative neoplasms

References

  1. The Leukemia & Lymphoma Society. Idiopathic myelofibrosis. 2007. Available at http://www.lls.org/#/diseaseinformation/myeloproliferativediseases/idiopathicmyelofibrosis. Accessed January 31, 2012.
  2. Morgan KJ, Gilliland DG. A role for JAK2 mutations in myeloproliferative diseases. Annu Rev Med. 2008;59:213-222.
  3. MedlinePlus Medical Encyclopedia, National Library of Medicine, National Institutes of Health. Myelofibrosis. Available at
    http://www.nlm.nih.gov/medlineplus/ency/article/000531.htm. Updated June 2, 2010. Accessed April 4, 2012.
  4. Mesa RA, Schwagera S, Radia D, et al. The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res. 2009;33:1199-1203.
  5. Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and Efficacy of JAK1 & JAK2 Inhibitor, INCB018424, in Myelofibrosis. New Eng J Med. 2010;16(363):1117-1127.

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