Peri-transplant ruxolitinib for patients with MF undergoing allo-HSCT

Results from a retrospective, single-center study evaluating the addition of peri-transplant ruxolitinib (periRux) to traditional graft-versus-host disease (GvHD) prophylaxis in 68 adults with myelofibrosis (MF) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) were published in Transplantation and Cellular Therapy by Mozaffari Jovein et al. The primary endpoint was GvHD-free relapse-free survival (GRFS). Secondary endpoints included overall survival (OS), non-relapse mortality (NRM), relapse rates, and engraftment.

Key data: Patients receiving periRux (n = 14) demonstrated a non-significant trend toward improved 1-year GRFS (51.0% vs 31.5%) and OS (92.86% vs 74.1%), as well as lower 1-year relapse rates (7.1% vs 13.8%) and NRM (7.1% vs 16.7%) compared with patients who did not receive periRux (n = 54). The overall prevalence of acute GvHD (aGvHD) and chronic GvHD (cGvHD) at 1 year was 33.8% and 27.9%, respectively. Grade III–IV aGvHD rates were similar between groups (28.6% vs 35.2%), whereas moderate-to-severe cGvHD at 1 year was lower in the periRux vs no periRux group (7.1% vs 33.3%). Clearance of driver mutations and/or complete donor chimerism (CC) was achieved in 84% of patients at Day 30 and Day 365.

Key learning: The addition of ruxolitinib to standard GvHD prophylaxis in patients with MF undergoing allo-HSCT appears feasible and is associated with favorable trends in relapse, survival, and moderate-to-severe cGvHD rates. These findings support evaluation in prospective, randomized trials.