Efficacy of Ruxolitinib in the management of chronic GVHD

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curative treatment for hematological diseases, with success rates improving due to advancements in conditioning regimens and new anti-graft versus host disease (GVHD) drugs. Ruxolitinib, an oral selective Janus kinase (JAK) 1 and 2 inhibitor has been used to mitigate the effects of various inflammatory and myeloproliferative syndromes, given the JAK kinase pathway's central role in cytokine signaling during inflammatory and immune processes. In this study we aimed to assess ruxolitinib's efficacy in patients with chronic GVHD (cGVHD).

This retrospective observational multi-center study involved 50 patients diagnosed with cGVHD after allo-HSCT in Turkey, who were treated with ruxolitinib between April 2018 and March 2024.

At the time of initiation of ruxolitinib treatment, most patients had severe cGVHD (n = 29, 58 %). The overall response rate at 6 months of ruxolitinib treatment was observed in 34 patients (68 %), including 6 patients (12 %) with complete responses and 28 patients (56 %) with partial responses, while 7 patients (14 %) experienced treatment failure. ECOG (2-4) performance status was established as an independent risk factor for adverse outcomes [p = 0.029, HR 3.492 (95 % CI: 1.139-10.705)]. At the two-year follow-up, the estimated survival rate was 52 %.

Ruxolitinib is safe and effective in the real-world setting for treating cGVHD, showing remission rates comparable to clinical trials. Further research with extended follow-up is necessary to confirm these findings, optimize dosing, and establish the best tapering strategies for responders.