LAUSR

Background Refractory chronic GVHD (cGVHD) is a common and highly lethal complication after allogeneic hematopoietic stem cell transplantation (HSCT). Cutaneous manifestations of this disease, such as scleroderma, strongly decrease quality of life. Mesenchymal Stem Cells (MSCs) expanded in vitro are involved in modulating immune responses in vivo, as well as tissue repair. Methods three patients with scleroderma-like severe chronic steroid-refractory GVHD were infused with 2 × 106 MSCs/KG of body weight divided in two doses. The MSCs were obtained from the bone marrow of non-matched donors. The patients were followed up through clinical and laboratory exams. Results All three patients showed complete response to treatment, with full recovery and no remaining sign of the disease. Mobility was significantly improved. One patient discontinued use of all immunosuppressive agents. All patients survived. Response lasted up to 311, 470 and 1792 days since infusion. Survival lasted 1737, 1329 and 3504 days. Conclusion Bone marrow-derived Mesenchymal stem cells was safe and effective treatment for scleroderma-like steroid-resistant chronic GVHD for these patients and should be evaluated in larger prospective trials.