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Treatment for Myelofibrosis

By

Allie Lemco Toren

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Treating Rare Blood Disorders

New advances in treatment for myeloproliferative diseases offer hope for people with these rare blood cancers.
ruxolitinib-myelofibrosis-cancer-drug

Myelofibrosis (MF) is a type of rare blood cancer that typically affects people over the age of 50. MF disrupts your body’s production of blood cells. In a healthy person, stem cells in the bone marrow mature over time into blood cells.

But if you have MF, these stem cells are abnormal and create scar tissue, which overtakes the bone marrow and prevents the production of blood cells. In some cases, the liver and spleen attempt to produce blood cells to compensate, but this causes them to enlarge.

These changes have many consequences for your health, which is why it’s important to treat MF as soon as you begin experiencing symptoms. Some treatments for MF focus specifically on relieving symptoms, while others aim to tackle the condition at the root. In fact, one treatment has actually been shown to cure the disease for some patients.

Not all patients with MF are symptomatic, so after you receive a diagnosis, your doctor may recommend watchful waiting and check in with you routinely. It’s possible to live symptom-free for years with myelofibrosis. But as soon as you begin experiencing symptoms, talk to your doctor immediately about treatment options.

Curing MF

A minority of patients with MF can be cured by receiving a stem cell transplant. In a transplant, a donor’s healthy stem cells are transferred to the patient to replace diseased stem cells; this brings the system back to normal. However, this procedure comes with lots of life-threatening risks, so it’s reserved for younger patients who may be better able to tolerate it. Sometimes it’s an option for patients who have tried every other treatment without success.

Treating MF Genetically

Researchers recently discovered that MF might be caused by problems with the JAK2 gene. This gene sends signals to help your bone marrow produce the right number of blood cells. But with MF, the JAK2 gene sends too many signals (called overactive signaling), creating scarring in the bone marrow and disrupting the healthy production of blood cells. We’re not sure why the JAK2 gene malfunctions in patients with MF, but researchers are exploring this in depth.

About 50% of MF patients have a specific defect in their JAK2 gene, and experts believe that all MF patients have some form of overactive signaling. The first medication approved by the U.S. Food and Drug Administration (FDA) to target the JAK2 gene is called ruxolitinib (Jakafi). This drug blocks the JAK2 gene from working, and has been shown to reduce the size of your spleen, relieve abdominal discomfort, and ease core symptoms of MF like bone pain, night sweats, and itching. We’re not sure yet if ruxolitinib can extend the life of MF patients, but clinical trials look promising.

Treating Anemia

If you have MF, your bone marrow doesn’t produce the right number and functionality of the three types of blood cells: red blood cells, white blood cells, and platelets. Red blood cells carry oxygen to the rest of your body, so when there’s not enough of them circulating, your body doesn’t get the oxygen it needs. This condition is called anemia, and it can cause symptoms like fatigue, shortness of breath, weakness, lightheadedness, headaches, irritability, and pale skin. Some anemic patients also experience fevers and night sweats. Anemia can negatively impact your prognosis and make your MF more severe.

To treat serious cases of anemia, doctors may turn to blood transfusions. But several medications are available that improve red blood cell counts. About 30% of MF patients have success after using male hormones called androgens. Experts are also studying the effects of new medications like thalidomide (Thalomid), lenalidomide (Revlimid), and pomalidomide (Pomalyst), which increase red blood cell levels and may also reduce the size of the spleen. In many cases, these drugs are combined with steroids. While clinical trials have shown success, these new medications come with a risk of serious birth defects and should be taken with caution.

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Medical Reviewers: William C. Lloyd III, MD, FACS Last Review Date: Jun 24, 2016

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Medical References

  1. Cervantes F. How I Treat Myelofibrosis. Blood. 2014;124(17): 2635-2642. http://www.bloodjournal.org/content/124/17/2635?sso-checked=true
  2. What Is Myelofibrosis? National Organization for Rare Disorders Physician Guides. http://nordphysicianguides.org/myelofibrosis/what-is-myelofibrosis/
  3. Primary Myelofibrosis. National Organization for Rare Disorders. http://rarediseases.org/rare-diseases/primary-myelofibrosis/
  4. Primary Myelofibrosis. MPN Research Foundation. http://www.mpnresearchfoundation.org/Primary-Myelofibrosis
  5. Myelofibrosis. Mayo Clinic. http://www.mayoclinic.org/diseases-conditions/myelofibrosis/basics/definition/con-20027210
  6. Primary Myelofibrosis Treatment & Management. Medscape. http://emedicine.medscape.com/article/197954-treatment#d11

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