Dr. Verstovsek on the Potential Utility of Luspatercept in Myelofibrosis

Srdan Verstovsek, MD, PhD, the United Energy Resources, Inc. Professor of Medicine, director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms​, and chief of the Section for Myeloproliferative Neoplasms in the Department of Leukemia of the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses the potential clinical utility of luspatercept-aamt (Reblozyl) in myelofibrosis.

Significant advances have been made in developing drugs to treat the symptoms of myelofibrosis, as well as splenomegaly, says Verstovsek. However, no treatment options are currently approved to treat myelofibrosis-related anemia, Verstovsek says.

As such, the potential integration of luspatercept to treat patients with myelofibrosis-related anemia is intriguing, Verstovsek says. In April 2020, the FDA approved luspatercept for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm (MPN) with ring sideroblasts and thrombocytosis.

Currently, luspatercept is being evaluated in the phase 3 INDEPENDENCE trial (NCT04717414) vs placebo in patients with MPN-associated myelofibrosis who are receiving concomitant JAK2 inhibitors and require blood transfusions, says Verstovsek. The purpose of adding luspatercept is to eliminate the need for blood transfusions in these patients whose symptoms are responding to JAK2 inhibition, Verstovsek concludes.