Off-Label Use of Ruxolitinib In Refractory Graft-Versus-Host Disease After Allogenic Stem Cell Transplantation


      Graft-versus-host disease (GVHD) is a severe complication of allogenic stem cell transplantation. Preliminary data suggest the potential role of ruxolitinib as a new treatment option in patients with corticosteroid-refractory GVHD. The aim of the present study was to explore the effectiveness and safety of ruxolitinib as a salvage therapy in this population.


      A single-center, retrospective, observational study was carried out. Inclusion criteria: all patients with corticosteroid-refractory GVHD treated with ruxolitinib until June 2016. Collected variables: age, sex, underlying disease, GVHD location, type (acute or chronic) and grade, previous therapies, Eastern Cooperative Oncology Group (ECOG) performance status at baseline, daily dose of ruxolitinib, treatment response and grade ≥3 adverse events (AE). Data sources: pharmacy information system and clinical charts review.


      13 patients met the inclusion criteria. 46% were male, with a median age of 46 years (range:32-51). The majority of patients (77%) had an ECOG performance status = 1. The most frequent underlying condition was myeloid acute leukemia. Most of the patients (77%) had more than one part of the body affected, being the most affected the cutaneous and gastrointestinal locations. GVHD was chronic in 62% of patients, and 54% had grade ≥ 3 GVHD. The median number of previous GVHD-therapies was 3 (range: 3-5). The most frequency therapies were corticosteroids, inmmunosuppression therapies with tacrolimus and rapamycin, and photopheresis. The median daily dose of ruxolitib was 20 (range: 5-25) mg. The overall response rate was 77% (complete response: 23%, partial response: 54%). Esophageal affectation in one patient was the only AE ≥3 recorded.


      Ruxolitinib appears to be an effective and safe treatment option for patients with GVHD who are refractory to corticosteroids and other available therapies. Randomized control trials are needed to confirm these promising results.