Myelofibrosis Clinical Trials 2023

What Are Myelofibrosis Clinical Trials?

Myelofibrosis is an uncommon type of blood cancer resulting in the bone marrow's buildup of scar tissue. Extensive scarring in the bone marrow disrupts the body’s normal production of healthy blood cells.

Over time, the liver and spleen start the production of blood cells. Because the liver and spleen are less effective at making blood cells, patients with myelofibrosis may develop anemia. It may result in the enlargement of the spleen due to the accumulation of red blood cells instead of releasing them into the body.

Myelofibrosis occurs due to genetic mutations in the bone marrow stem cells. Scientists do not know what leads to these genetic mutations, requiring extensive myelofibrosis clinical trials to develop effective diagnoses, treatments, and prevention plans.

Why Is Myelofibrosis Being Studied Through Clinical Trials?

Myelofibrosis clinical trials can help researchers explore more effective ways of treating the condition and managing the side effects of associated medications. Pioneering research into myelofibrosis can identify new medication options that are currently needed. Most older patients are not eligible for marrow transplants, which makes myelofibrosis clinical trials all the more necessary.

The current body of research is focused on interleukin-1 (IL-1) in patients. Elevated levels of IL-1 may result in the inflammation of harmful mutations in the bone marrow. Further research in this area could provide helpful information for treating myelofibrosis.

What Are The Types of Treatments Available For Myelofibrosis?

No treatment can cure myelofibrosis in patients. The only reliable cure for myelofibrosis is an allogeneic stem cell transplant, which is risky for some patients, including older individuals and those with health problems. Treatment of myelofibrosis depends on various factors. A large interindividual variability in patient symptoms requires different treatment options.

Some patients may not require immediate treatment for myelofibrosis because they remain symptom-free for years. All patients will eventually require close monitoring. Doctors may prescribe medication to lower blood cell count to relieve patient symptoms. Common medications include Ruxolitinib, Hydroxyurea, and Interferon alfa.

What Are Some Recent Breakthrough Clinical Trials For Myelofibrosis?

The following studies indicate progress in diagnosing, treating, and managing Myelofibrosis:

2015 - Efficacy of Fedratinib for Myelofibrosis - This study evaluates the safety and efficacy of fedratinib, a JAK2-selective inhibitor for treating primary and secondary myelofibrosis. The clinical trials found that oral fedratinib reduced splenomegaly in patients with myelofibrosis.

2021: Emerging Drugs for Treating Myelofibrosis - MF patients with disease-related cytopenias are ineligible for JAK2 inhibitors. However, this clinical trial looks at pacritinib and momelotinib to modify disease progression.

Who Are Some Of The Key Opinion Leaders / Researchers / Institutions Conducting Depression Clinical Trial Research?

Douglas A Tremblay is an Assistant Professor of Medicine at the Icahn School of Medicine at Mount Sinai. His research areas include acute myeloid leukemia and myeloproliferative overlap syndromes. He has published over 45 articles on myeloproliferative, including serving as an investigator in several clinical trials. He is a leading authority on myelofibrosis research.

Ann Mullally is an associate professor of medicine at Harvard Medical School and a leading cancer researcher in the field of myeloid malignancies. She has published several breakthrough research in myelofibrosis, including the review of murine models for the characterization of MF pathobiology.