Real-world experience with ruxolitinib for steroid-refractory acute graft-versus-host disease: a single center experience

Abstract

Steroid-refractory acute graft-versus-host disease (SR-aGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ruxolitinib (RUX), an oral JAK1 and JAK2 inhibitor, has recently been approved for patients with SR-aGVHD. The aim of this study was to evaluate RUX efficacy and toxicity in a real-world setting. Eighteen patients received RUX at 5 mg or 10 mg twice a day after a median 3 lines of prior unsuccessful immunosuppressive therapy. Median time on RUX therapy was 28 days (range 7–129). Five patients (28%) responded to RUX, including 4 complete responses and 1 partial response. Response to RUX was irrespective of aGVHD grade and the number of involved organs. One-year overall survival (OS) was 60% for RUX-responders versus 31% for non-responders (p = ns). Treatment duration greater than 29.5 days was found to have a positive impact on OS (p < 0.007). Major adverse events during RUX treatment were grade 3–4 thrombocytopenia (61% of patients) and cytomegalovirus reactivation (50%). After median follow-up of 55 days (range 29–706), 14 patients (78%) died, mainly due to further progression of GVHD. RUX may represent a valuable therapeutic option for some patients with advanced SR-aGVHD, but more studies are warranted.

Basiliximab Treatment for Patients With Steroid-Refractory Acute Graft-Versus-Host Disease Following Matched Sibling Donor Hematopoietic Stem Cell Transplantation

“…These findings are similar with previous studies (ie, ORR at any time: 65%–70% ORR at day 28: 54%–59% 21 , 54 , 66 ). In addition, we found ORR at any time was ranging from 16.7% to 94% 50 , 52 for other IL-2RA, 28%–81.5% 35 , 36 , 64 , 67 for ruxolitinib, 30.8%–79% 40 , 65 , 68 for MMF, 59% 59 for ATG, 28%–67% 44 , 68 , 69 for TNF blockades, and 46.6%–58.7% 62 , 70 , 71 for MSCs. Hence, data from this analysis support that this approach has a beneficial effect to SR-aGVHD patients after MSD-HSCT.…”