Emerging Therapies in Myelofibrosis: Highlights From SOHO 2020

Abstract

S everal clinical trials investigating novel agents and combinations in myelofibrosis (MF) are showing promise. Such data have implications for advanced practitioners (AP) managing disease-related symptoms associated with splenomegaly, constitutional symptoms, and symptomatic anemia. At present, two Janus kinase inhibitors (JAKi) are U.S. Food & Drug Administration approved for frontline MF: ruxolitinib (Jakafi) and fedratinib (Inrebic). However, patients with MF who are red blood cell (RBC) transfusion dependent, either progress on or are resistant to or intolerant to ruxolitinib, have disease-related cytopenias such as anemia or thrombocytopenia, or have high-risk disease, continue to have unmet needs. An emerging focus is on exploring novel agents or ruxolitinib-based combinations that may augment disease responses by targeting alternative pathways to yield more favorable outcomes in subpopulations.