Dr Hunter on Luspatercept and Other Treatment Options for Anemia in Myelofibrosis

Anthony M. Hunter, MD, an associate professor in the Department of Hematology and Medical Oncology at Emory University School of Medicine, medical director of the Immediate Care Center, and leader of the Myeloproliferative Neoplasm Program at Emory Winship Cancer Institute, discussed treatment decision-making for the management of anemia in patients with myelofibrosis.

Hunter emphasized the limited number of effective treatment options currently available for anemia management in patients with myelofibrosis. Although luspatercept-aamt (Reblozyl) and erythropoiesis-stimulating agents (ESAs) are not formally FDA approved for the treatment of anemia in patients with myelofibrosis, Hunter noted that data from earlier studies and their inclusion in National Comprehensive Cancer Network (NCCN) Guidelines have increased physician confidence in their use and improved payer support for reimbursement. He highlighted that the NCCN Guidelines are particularly useful for clinicians who may treat myelofibrosis less frequently and are seeking additional therapeutic strategies for anemia management.

Hunter explained that anemia remains a major unmet need for patients with myelofibrosis, with currently available therapies offering modest and often incomplete benefit. Traditional approaches such as ESAs can have limited efficacy, particularly in patients with elevated baseline erythropoietin levels. Given the lack of robust, approved options, clinicians are increasingly utilizing agents like luspatercept to improve anemia outcomes and reduce transfusion burden in practice.

In terms of future treatment directions, meaningfully modifying the underlying disease biology could serve as a way to address anemia in patients with myelofibrosis. Current approaches, including ESAs and luspatercept, primarily target anemia itself rather than the malignant clone driving the disease. Hunter noted that anemia responses and transfusion independence observed in JAK inhibitor studies have been associated with improved long-term survival outcomes, but established JAK inhibitors such as ruxolitinib (Jakafi) do not substantially reverse bone marrow pathology or prevent leukemic transformation.

Hunter expressed optimism regarding JAK inhibitor–based combination strategies designed to target multiple disease pathways simultaneously. The broader goal of these approaches is true disease modification, potentially restoring marrow function and improving anemia as a downstream consequence of more effectively suppressing the underlying disease.