Autologous peripheral hematopoietic stem-cell transplantation in a patient with refractory pemphigus

The aim of this study is to explore the effective-ness of autologous peripheral hematopoietic stem-cell transplantation in the treatment of refractory pemphigus.A 35-year-old male patient presented with a 4-year history of recurrent bullae on his trunk and extremities.The dia-gnosis of pemphigus was made on the basis of the clinical,histologic and immunofluorescence findings.The patient had shown resistance to conventional therapy with gluco-corticoid and immunosuppressive agents.Two months before admission,he complained of hip joint pain.X-ray and CT scan revealed aseptic necrosis of the femoral head.Stem-cell mobilization was achieved by treatment with cyclophosphamide,granulocyte colony-stimulating factor(G-CSF)and rituximab.Peripheral blood stem cells were collected via leukapheresis and cryopreserved for later use.Immunoablation was accomplished by using cyclophospha-mide(200 mg/kg;divided into 50 mg/kg on days-5,-4,-3,and-2),antithymocyte globulin(ATG;10 mg/kg;divided into 2.5 mg/kg on days-6,-5,-4,and-3),and rituximab(1200 mg/d;divided into 600 mg/d on days 0 and 7).Autologous peripheral hematopoietic stem cell transplanta-tion was followed by reconstitution of the immune system which was monitored by flow cytometry.The glucocorti-coid was withdrawn immediately after transplantation.The pemphigus titer turned negative 6 weeks after transplanta-tion and remained negative.The patient was in complete drug-free remission with no evidence of residual clinical or serological activity of pemphigus during 1 year of follow-up.The patient’s response suggests that autologous peri-pheral hematopoietic stem cell transplantation may be a potential“cure”for refractory pemphigus.However,fur-ther studies are needed to evaluate the risk-benefit ratio of this approach in patients with pemphigus showing resist-ance to conventional therapy.