Steroid-refractory chronic graft-versus-host disease (cGVHD) is associated with high morbidity. To date, there is no standard therapy for patients who fail to respond to steroids.
In this study, we evaluated the efficacy and safety of imatinib mesylate and mycophenolate mofetil (MMF) to treat sclerotic/fibrotic type cGVHD.
design: In this non-randomized, open-label, single-arm, multicenter prospective phase II study, we enrolled patients with steroid-refractory sclerotic/fibrotic type cGVHD. The primary endpoint was the overall response rate to imatinib mesylate plus MMF in one year and the secondary endpoints included safety, quality of life, discontinuation of steroids, and the overall survival rate.
Overall, 13 patients were enrolled, with a median age of 10.4 years (range, 5.0-20.1 years). All patients received a myeloablative conditioning regimen. Specifically, six of these patients had previously experienced acute GVHD. The most frequently affected organs were the eyes, lungs, skin, and liver. There were two premature deaths. One patient died of pulmonary infection and progression of cGVHD; another patient died from neuroblastoma progression and septic shock. The overall response rate was 76.9% (10/13 patients) and the median dose of steroid decreased from 1.0 mg/kg/day to 0.21 mg/kg/day. Finally, the one year overall survival rate was 84.6% (n=13), while the common adverse events included liver enzymes and serum creatinine elevation, and fever.
Although there was a limited sample size, imatinib mesylate plus MMF treatment for cGVHD disease demonstrated promising results with acceptable toxicity. This study was registered at www.clinicaltrials.gov (#NCT01898377).

Copyright © 2021. Published by Elsevier Inc.

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