Myelofibrosis (PMF) Clinical Trials and Treatments

Myelofibrosis is a myeloproliferative neoplasm characterized by excessive fibrosis (scarring) in bone marrow, leading to anemia, constitutional symptoms, splenomegaly, and increased risk of transformation to acute leukemia. The disease results from clonal proliferation of hematopoietic stem cells, most commonly driven by JAK2, CALR, or MPL mutations. These mutations cause aberrant cytokine signaling, leading to excessive production of fibrogenic cytokines that promote bone marrow fibrosis.

The most common symptoms of Myelofibrosis include Fatigue and weakness from anemia, Abdominal discomfort from enlarged spleen, Shortness of breath, Easy bruising and bleeding, Night sweats and fever, among others. Approximately 0.5-1.5 cases per 100,000 people; median age of diagnosis is 65 years. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Myelofibrosis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. The FDA has now approved 4 JAK inhibitors for myelofibrosis: ruxolitinib (Jakafi) in November 2011, fedratinib (Inrebic) in August 2019, pacritinib (Vonjo) in February 2022 and momelotinib (Ojjaara) in September 2023. Vonjo is approved for people whose platelet count is below 50,000, under an accelerated approval whose continuation may depend on a confirmatory trial, and Ojjaara is approved for people with anemia.

Yes. As of September 2026, Trial Friend lists 2 FDA-approved drugs used to treat Myelofibrosis: Vonjo (pacritinib), first approved in 2022; and Ojjaara (momelotinib), first approved in 2023.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Myelofibrosis arises from acquired (somatic) genetic changes in the affected cells, commonly involving JAK2, CALR and MPL, rather than from an inherited variant. Driver mutations are acquired in blood-forming cells and are not inherited; JAK2, CALR and MPL testing on blood or marrow is standard for diagnosis. Approximately 0.5-1.5 cases per 100,000 people; median age of diagnosis is 65 years.

For Myelofibrosis the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA, looking for JAK2, CALR and MPL. Driver mutations are acquired in blood-forming cells and are not inherited; JAK2, CALR and MPL testing on blood or marrow is standard for diagnosis.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for Myelofibrosis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Myelofibrosis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.