title: A case of sclerodermatous graft-versus-host disease responsive to imatinib therapy.
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-β 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.
authors.affiliation: Department of Dermatology, University of Wisconsin, Madison, Wisconsin 53715, USA.
authors.forename: Jozef
authors.initials: J
authors.lastname: Lazar
authors.forename: Tasneem
authors.initials: T
authors.lastname: Poonawalla
authors.forename: Joyce M C
authors.initials: JM
authors.lastname: Teng
mesh_terms: D000293:Adolescent; D001549:Benzamides; D005260:Female; D006086:Graft vs Host Disease / Q000150:complications* / Q000188:drug therapy* / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D000068877:Imatinib Mesylate; D010879:Piperazines / Q000008:administration & dosage*; D047428:Protein Kinase Inhibitors / Q000008:administration & dosage; D011743:Pyrimidines / Q000008:administration & dosage*; D012595:Scleroderma, Systemic / Q000188:drug therapy* / Q000209:etiology* / Q000276:immunology
chemical_list: D001549:Benzamides; D010879:Piperazines; D047428:Protein Kinase Inhibitors; D011743:Pyrimidines; D000068877:Imatinib Mesylate