Dr. Verstovsek on the Use of Luspatercept to Treat Anemia Associated With 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 use of luspatercept (Reblozyl) to treat anemia associated with myelofibrosis.

Several challenges must be considered when treating patients with myelofibrosis, including a short life expectancy of less than 5 years, Verstovsek explains. Also, a limited number of agents have been approved to treat symptoms associated with the disease, such as enlarged spleen, poor quality of life, and anemia. JAK inhibitors are currently being used to treat splenic symptoms, such as ruxolitinib (Jakafi) and fedratinib (Inrebic), Verstovsek says.

Although no agents have received regulatory approval to treat anemia in this patient population, luspatercept may represent a solution, Verstovsek notes. Luspatercept was approved by the FDA in April 2020 for myelodysplastic syndrome (MDS)-associated anemia, including subtypes such as MDS with myelofibrosis ring sideroblasts and myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.

Currently, luspatercept is being studied in patients with myelofibrosis, which is similar in nature to MDS, and it's possible that the agent will show activity in both disease types because of this, Verstovsek concludes.