Following its 2011 approval for myelofibrosis (MF), ruxolitinib has become a cornerstone in treating MF and polycythemia vera (PV), two common myeloproliferative neoplasms (MPNs). Despite its benefits, some patients may not respond or may discontinue treatment due to various reasons. Recent studies presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024 have explored ruxolitinib in combination with other drugs to enhance treatment efficacy.
MANIFEST-2 Trial: This phase 3 study evaluated pelabresib, a BET inhibitor, combined with ruxolitinib in JAK inhibitor-naive MF patients. Results showed a significant reduction in spleen volume and improvement in symptom scores compared to ruxolitinib alone, with benefits maintained over 72 weeks.
Bomedemstat Study: An ongoing phase 2 trial investigates bomedemstat, an LSD1 inhibitor, with ruxolitinib in advanced MF patients. Preliminary results indicate improvements in symptom scores and spleen volume reduction, with a favorable safety profile.
RESTORE Trial: This phase 2 study examines elritercept, with or without ruxolitinib, in MF patients with anemia. Early data suggest potential benefits in spleen volume reduction, symptom improvement, and addressing cytopenias, with ongoing evaluations of safety and efficacy.
These studies underscore the ongoing efforts to improve outcomes for MPN patients through innovative combination therapies, offering hope for those who may not respond to current treatments.
