New Medication Alleviates Severe Anemia for Bone Marrow Disorder

Mayo Clinic researchers have found a new medication, pomalidomide, designed to optimize immune functions for patients with myelofibrosis, a serious bone marrow disorder. According to a Mayo Clinic study presented Monday at the 50th Annual Meeting of the American Society of Hematology in San Francisco, pomalidomide is well tolerated in patients with myelofibrosis.

Myelofibrosis is a form of blood cancer that disrupts the body’s normal production of blood cells, especially red blood cells. The disease is associated with extensive scarring of the bone marrow. The major clinical manifestations of myelofibrosis include severe anemia — causing weakness and fatigue — and enlargement of the spleen and liver — causing difficulty eating and abdominal pain and discomfort.

Pomalidomide is the latest in the class of immunomodulatory agents that includes thalidomide and lenalidomide. Previous research found thalidomide and lenalidomide effective for alleviating severe anemia in approximately 20% of patients with myelofibrosis. However, the value of these drugs is undermined by their respective potential to cause peripheral neuropathy (nerve degeneration) and severe myelosuppression (low white blood cell and platelet count).

“Pomalidomide provides good promise for myelofibrosis patients who suffer from anemia,” says Ayalew Tefferi, M.D., hematologist and lead researcher on the study. “This well tolerated treatment has the potential to help at least one-third of patients with anemia and myelofibrosis.”

Thus far, the study have shown that approximately one third of patients taking the study drug have become red blood cell transfusion independent or experienced a more than 2 g/dL increase in their hemoglobin level.

This clinical trial was sponsored by Celgene.

In addition, Dr. Tefferi and Animesh Pardanani, M.B.B.S., Ph.D., are also the lead investigators of a Phase I clinical trial that is evaluating the toxicity and efficacy of a novel small molecule inhibitor of JAK2 in myelofibrosis patients. For more information on this Phase I study, please view the abstract.

Dr. Tefferi’s Phase I clinical trial was sponsored by TargeGen.

Primary myelofibrosis is also referred to as agnogenic myeloid metaplasia or idiopathic myelofibrosis. An uncommon disease, myelofibrosis can occur at any age, although it most frequently develops after age 50. The disease is currently believed to arise from an acquired cancer-causing genetic mutation whereas the scarring (fibrosis) of the bone marrow is thought to be a reaction to the neoplastic proliferation of blood cells.

For more information on hematology research, visit the Mayo Clinic Cancer Center.