Limited efficacy and tolerance of imatinib mesylate in steroid-refractory sclerodermatous chronic GVHD

摘要

Imatinib mesylate (IM), a tyrosine kinase inhibitor, has shown efficacy for the treatment of chronic GVHD (cGVHD), with overall response rates of fibrotic skin symptoms evaluated in 2 open-label studies ranging from 50% to 79%. To assess the global long-term effectiveness of IM for sclerodermatous cGVHD, we performed a retrospective study on 39 patients with steroid-resistant disease followed in 6 French hospitals (Table 1). At the onset of IM, all patients had sclerodermatous grade 2 (n = 6, 15%) or grade 3 cGVHD (ie, deep sclerotic features, fasciitis, skin ulcers, or involvement of more than 50% of the body surface area; n = 33, 85%) according to the National Institutes of Health (NIH) grading. Twenty-five patients (64%) had 1 or more organs involved apart from the skin, and bronchiolitis obliterans was observed in 18 patients (46%). Thirty-seven patients (95%) had received 2 or more treatment lines for cGVHD before IM treatment. IM was started after a mean delay of 29 ± 28 months after the diagnosis of cGVHD.