The Evolving Role of Targeted Therapy in Myelofibrosis

In the second installment of an expert roundtable series on myelofibrosis moderated by Prithviraj Bose, MD, of The University of Texas, MD Anderson Cancer Center, experts dive into the current treatment landscape and ongoing clinical research shaping the future of care. The panel includes Naseema Gangat, MBBS of Mayo Clinic,  Pankit J. Vacchani, MD of the University of Alabama at Birmingham, and Idoroenyi Amanam, MD of City of Hope.

The therapeutic landscape for myelofibrosis is evolving. The experts’ discussion focuses on the significant advancements in JAK inhibitor treatments and new investigational agents. The conversation opens with an overview of the transformative impact ruxolitinib has had since its FDA approval in 2011. As the first JAK1/JAK2 inhibitor available, ruxolitinib has shown dramatic improvements in spleen size and symptom relief, as well as a survival benefit validated in both clinical trials and real-world studies. However, challenges such as cytopenias and dose-dependent efficacy are emphasized, highlighting the need for careful dose management.