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Patient-Centered Approaches in Today’s MF Treatment Paradigm

By Pankit Vachhani, MD, Prithviraj Bose, MD, Idoroenyi Amanam, MD, Naseema Gangat, MBBS - Last Updated: May 12, 2025

In this roundtable discussion moderated by Prithviraj Bose, MD of The University of Texas MD Anderson Cancer Center, leading hematologists/oncologist explore patient-centered approaches within the current treatment paradigm for myelofibrosis (MF). Panelists 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 highlight ongoing clinical trials and the evolving therapeutic landscape.

Guiding this portion of the conversation, Dr. Vacchani beings by providing insights on the phase 3 INDEPENDENCE study evaluating the addition of luspatercept to ruxolitinib in patients requiring transfusions. He underscores promising results from the phase 2 EMBARK trial, where a higher dose of imetelstat showed a nearly doubled median overall survival in relapsed/refractory MF patients. The ongoing phase 3 IMpactMF trial will further assess its second-line potential.

Dr. Gangat chimes in, introducing DISC-0974, an anti-hemojuvelin antibody that suppresses hepcidin, addressing elevated levels seen in MF. This agent shows promise in monotherapy and in combination with JAK inhibitors. She also contrasts it with luspatercept, noting differing efficacy in MDS and MF due to distinct disease pathogenesis—iron-restricted vs. ineffective erythropoiesis.

The talk continues into how these emerging therapies not only provide potential symptomatic relief but also reflect a deeper understanding of disease mechanisms, opening doors to more tailored, patient-centered care strategies. They also discuss luspatercep in combination with momelotinib in the ODYSSEY trial, and the investigational use of elritercept, another receptor ligand trap with origins linked to sotatercept.

The panel of experts emphasizes a shift in the treatment paradigm that balances clinical response with quality of life and anemia support, especially in patients on JAK inhibitors, paving the way for improved outcomes for patients with MF.