Abstract

Acute graft-versus-host disease (aGVHD) is a life-threatening complication that can develop after allogeneic hematopoietic stem cell transplantation. Patients with steroid-refractory aGVHD (SR-aGVHD) have an extremely poor prognosis. Ruxolitinib is an approved treatment for SR-aGVHD. However, there is a paucity of real-world data on the clinical outcomes of patients with SR-aGVHD treated with ruxolitinib.

We conducted a retrospective analysis using hospital records of the clinical outcomes of patients who underwent stem cell transplantation at our center between January 2021 and December 2022 and developed steroid-refractory aGVHD which was treated with ruxolitinib.

During the study period, 381 patients underwent allogeneic stem cell transplantation at our center. Amongst these, 160 (42.0%) developed aGVHD. Of these, 59 (36.8%) had SR-aGVHD and 40 were treated with ruxolitinib. Ruxolitinib therapy was administered after a median of 6 days (range 3-29) from onset of aGVHD. Amongst the 28 patients who survived at day 28 (12 died before the day 28 response could be assessed), a total of 16 patients (57.1%) attained a response (complete response (n=12) or partial response (n=4)). Infectious complications were the most common adverse event (n=39; 97.5%), followed by severe cytopenia (grades 3 to 4) in 25 (62.5%) patients. The median follow-up of the cohort was 5 months (range 1 to 29 months). At the last follow up, 30 (75%) patients died; 3 patients died of progression of steroid-refractory aGVHD, 14 died of progression of aGVHD with infection, 10 died of underlying infection and 3 had disease relapse.

From our real-world analysis, we conclude that though the outcomes in patients with SR-aGVHD responding to ruxolitinib are encouraging, there is still a large unmet need for novel strategies for improving outcomes and reducing infection-related mortality, even while there is access to ruxolitinib.