Imatinib Mesylate for the Treatment of Steroid-Refractory Sclerotic-Type Cutaneous Chronic Graft-versus-Host Disease

Kristin Baird, Leora E. Comis, Galen O. Joe, Seth M. Steinberg, Fran T. Hakim, Jeremy J. Rose, Sandra A. Mitchell, Steven Z. Pavletic, William D. Figg, Lawrence Yao, Kathleen C. Flanders, Naoko Takebe, Stefanie Sarantopoulos, Susan Booher, Edward W. Cowen*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

46 Scopus citations


Sclerotic skin manifestations of chronic graft-versus-host disease (ScGVHD) lead to significant morbidity, including functional disability from joint range of motion (ROM) restriction. No superior second-line therapy has been established for steroid-refractory disease. Imatinib mesylate is a multikinase inhibitor of several signaling pathways implicated in skin fibrosis with in vitro antifibrotic activity. We performed an open-label pilot phase II trial of imatinib in children and adults with corticosteroid-refractory ScGVHD. Twenty patients were enrolled in a 6-month trial. Eight received a standard dose (adult, 400 mg daily; children, 260 mg/m2 daily). Because of poor tolerability, 12 additional patients underwent a dose escalation regimen (adult, 100 mg daily initial dose up to 200 mg daily maximum; children, initial dose 65 mg/m2 daily up to 130 mg/m2 daily). Fourteen patients were assessable for primary response, improvement in joint ROM deficit, at 6 months. Primary outcome criteria for partial response was met in 5 of 14 (36%), stable disease in 7 of 14 (50%), and progressive disease in 2 of 14 (14%) patients. Eleven patients (79%), including 5 with partial response and 6 with stable disease, demonstrated a positive gain in ROM (range of 3% to 94% improvement in deficit). Of 13 patients with measurable changes at 6 months, the average improvement in ROM deficit was 24.2% (interquartile range, 15.5% to 30.5%; P = .011). This trial is registered at as NCT007020689.

Original languageEnglish
Pages (from-to)1083-1090
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Issue number6
StatePublished - Jun 2015
Externally publishedYes


  • Allogeneic hematopoietic stem cell transplantation
  • Fasciitis
  • Graft-versus-host disease
  • Imatinib mesylate
  • Sclerosis


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