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Myelofibrosis Clinical Trials

Myelofibrosis Clinical Trials

Myelofibrosis Clinical Trials provide patients with early access to innovative and non-traditional treatment options such as immunotherapy, targeted therapy, and chemotherapy. Clinical trials explore new ways to prevent, detect, or treat diseases.

Myelofibrosis Clinical Trials are extremely important to further our knowledge of this disease. Myelofibrosis usually develops slowly. In the very early stages of the disease, many people do not experience signs or symptoms. As the deterioration of normal blood cell production increases, signs and symptoms are often experienced.

What Are Myelofibrosis Clinical Trials?

Many exciting new treatments are currently being tested through clinical trials run by experts. Research is expanding rapidly with a focus on drug types such as:

  • Alternative JAK inhibitors
  • Transfusion addiction agents
  • Antifibrinolytic agents
  • Telomerase inhibitors
  • BET inhibitors

New strategies are being investigated by trying many drug combinations in clinical trials. These studies give hope for the future treatment of the disease. Clinical trials are a great alternative for patients who are not benefiting from other treatments.

Does Myelofibrosis Turn into Leukemia?

Myelofibrosis is a rare disease and can be seen in both men and women. People of all ages can have myelofibrosis, but it is more often diagnosed in people over the age of 50. The diagnosis of myelofibrosis is usually seen before the age of 3 in children.

Myelofibrosis, a rare type of blood cancer, is considered a type of chronic leukemia. Acute leukemia may develop in some patients with myelofibrosis. Acute myelogenous leukemia, a type of rapidly progressing blood and bone marrow cancer, is one of the complications of myelofibrosis.

Other complications that may arise from myelofibrosis include increased blood pressure in the liver (portal hypertension), abdominal and back pain due to enlargement of the spleen, and bleeding in the gastrointestinal system due to tumor formation.

Myelofibrosis Targeted Treatments

In 2005, the discovery of JAK2 mutations enabled the development of a targeted therapy for patients with myelofibrosis. The JAK1/JAK2 inhibitor, ruxolitinib, is the first FDA-approved drug for the treatment of myelofibrosis. Fedratinib is another drug that has also been approved as the first treatment option for high-risk myelofibrosis.

Interferon is an immune therapy that helps reduce the excess of unhealthy blood cells and cytokines that cause fibrosis in the bone marrow. However, there may be significant side effects of interferon that may be difficult to tolerate for some patients. If you are looking for alternative treatment options, it is important to consult your doctor before making any decisions.


  • Maureen says:

    I was hoping to try Pegasys but dr says it’s not fda approved for MF. Are there exemptions?

    • Cancer Treatment Expert says:

      Hi Maureen, thank you for your question, many drugs are currently in the clinical trial process, they are not yet FDA approved but patients can access them through clinical trials, we would be happy to discuss all available clinical trials with you

  • Maureen Kleiman says:

    Looking to try Pegasys. I’m MF 3 on jakafi 5/5 reduced spleen from 24-to 17 CM. Dr tried to double dose and it was quickly overwhelming and stopped for 2 days. Back on5/5 now .
    I’m m 74 , my numbers aren’t terrible – worst number is wbc -35. Other numbers mostly close to low normal. I declined SCT due to fact I overreact to pharmaceuticals, had horrible side effects on HU. Any Pegasys trials ? I’m in M Fl between Gainesville and Jacksonville. My daughter lives in Coral Springs near Ft Lauderdale.

  • D e n a says:

    Hi good morning my name is Dina and I have been diagnosed with thrombocytosis the high platelet condition several years later a secondary myelofibrosis right now I’m being treated with the jakafi medication and also Hydrox at the same time my platelets are still screaming High over million that counts are up I’m anemic the hemoglobin and hemo crepes low I’m not really too sure mixing the jakafi in the hydroxyurea together was a good idea it’s been over 90 days now and blood work is completely out of whack

    • Cancer Treatment Expert says:

      We are sorry to hear of the difficult time you are experiencing. We may have a clinical trial that you could qualify for given that you are currently receiving Jakafi. One of our experienced patient advocates will reach out to you to discuss your options.

  • Sam Yeboa says:

    I have Myelofibrosis and have tried all the available medicines including jakafi, busulfan, interferon, hydroxyurea and anagrelide but platelets are still high in the millions. Please let me know if there’s any help for me. Thanks

    • Cancer Treatment Expert says:

      Hi Sam,

      We are sorry to hear of the difficulties you are experiencing with progression of your disease. There are many clinical trials available that could provide benefit and possibly slow the progression of your disease. We would be happy to review those options with you. Please reach out to our Patient Advocates at 844-627-7246 to get started or submit a request at

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