October 1, 2022
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What Is Myelofibrosis? Symptoms, Causes, Diagnosis, Treatment, and Prevention

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Your treatment approach will depend on how advanced your cancer is, your overall health, what genetic mutations you have, if any, and other factors.

“Myelofibrosis is very heterogenous [diverse],” explains Mesa. “Plans need to be quite individualized because not everybody is the same.”

For example, a small subset of people with myelofibrosis who are low risk and minimally affected by the disease may be candidates for the simplest treatment of all: observation. This means the doctor will watch the cancer carefully, but the patient isn’t on any active treatment.

Medications for Myelofibrosis

Currently, FDA-approved medicines to treat myelofibrosis include:

  • Ruxolitinib (Jakafi)
  • Fedratinib (Inrebic)
  • Pacritinib (Vonjo)
These medicines are all JAK-inhibitors, which work by blocking the processes of faulty genes.

Medications for Anemia

Patients with myelofibrosis who develop anemia (a low red blood cell count) might be candidates for additional treatments, such as:

  • Chemotherapy
  • Steroids
  • Androgens (synthetic hormones)
  • Immunomodulators, such as thalidomide or lenalidomide
  • Interferon (specific proteins that help the body fight infections)
  • A blood transfusion

Treatments for an Enlarged Spleen

An enlarged spleen due to myelofibrosis might be treated with one or more of the following:

  • A JAK inhibitor
  • Chemotherapy
  • Radiation
  • Surgery to remove the spleen
  • Interferon

Stem Cell Transplant for Myelofibrosis

A stem cell transplant, also called a bone marrow transplant, is an option for some people with myelofibrosis. This involves replacing diseased bone marrow with healthy blood stem cells from a donor.

Candidates for a stem cell transplant are usually younger, healthy patients.

Before undergoing the transplant, the patient receives chemotherapy or radiation to destroy the diseased bone marrow. Stem cells from a compatible donor are then given via an infusion.

A stem cell transplant has the potential to cure myelofibrosis, but the procedure can cause life-threatening risks.

Because of these risks, Mesa says stem cell transplants are performed in less than 10 percent of patients with myelofibrosis.

“A stem cell transplant can, even in the best circumstances, have a 10 to 15 percent chance of fatal complications,” Mesa says. “To put that in perspective, open-heart surgery carries only a 1 percent risk.”

Clinical Trials

Participating in a clinical trial may be an option for some people with myelofibrosis. These studies might allow you to receive a therapy that isn’t yet available to the public.

Mesa says there are several drugs being studied in clinical trials that could be beneficial for people with myelofibrosis.

“Be hopeful. There are a lot of new medicines in development, a lot of things that are being discovered, and a lot of people out there rooting for you even though this is a rare cancer,” he says.



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