A Case of Sclerodermatous Graft-versus-Host Disease Responsive to Imatinib Therapy PEDIATRIC DERMATOLOGY Lazar, J., Poonawalla, T., Teng, J. M. 2011; 28 (2): 172-175

Abstract

Sclerodermatous graft-versus-host disease (sGVHD) is a rare, late complication of hematopoietic cell transplantation. Classified as a variant of chronic graft-versus-host disease, sGVHD is thought to be predominantly an immune-mediated response characterized by aberrant T-cell function and dysregulation of tyrosine kinase cascades. Recently, the profibrotic cytokine transforming growth factor B and stimulatory autoantibodies against the platelet-derived growth factor receptor have been implicated in the pathogenesis of sGVHD. Treatment of sGVHD remains disappointing and largely limited by systemic side effects. Imatinib mesylate is a small molecule tyrosine kinase inhibitor that has been shown to selectively inhibit both the platelet-derived growth factor receptor and transforming growth factor-ß signaling pathways. We report a case of sGVHD in a pediatric patient that was resistant to traditional therapy but showed improvement in cutaneous symptoms following daily treatment with 400 mg of imatinib mesylate. Due to its favorable side-effect profile, specificity for molecular pathways deranged in sGVHD and proven efficacy in other sclerodermoid diseases, imatinib mesylate is a promising new tool in the management of this challenging disease.

View details for DOI 10.1111/j.1525-1470.2010.01301.x

View details for Web of Science ID 000290381900017

View details for PubMedID 21504445