Abstract: Objective To explore the possibility of autologous peripheral hematopoietic stem cell transplantation in treatment with refractory pemphigus. Patient and Intervention A 35-year-old male patient presented with a 4-year history of recurrent bullae on his trunk and extremities. The diagnosis of pemphigus was made on the basis of the clinical, histologic, and immunofiuorescence findings. The patient showed resistance to conventional therapy with glucocorticoid and immunosuppressive agents. Two months before admission, he complained of hip joint pain, and X-ray and CT scan revealed amicrobic 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 using cyclophosphamide(200mg/kg; divided 50 mg/kg on days -5,-4,-3, and -2),antithyrnocyte globulin(ATG; 10 mg/kg; divided 2.5mg/kg on days -6,-5,-4, and -3),and rituximab(1200 mg/d; divided 600mg/d on days 0 and 7).Autologous peripheral hematopoietic stem cell transplantation was followed by reconstitution of the immune system, which was monitored by flow cytometry. Results The glucocorticoid was withdrawn immediately after transplantation. His pemphigus titerturned negative at 6 weeks posttransplantation and remained negative. The patient was in complete drug-free.remission with no evidence for residual clinical or serological activity of pemphigus during 1 year of follow-up. Conclusions The patient's response suggests that autologous peripheral hematopoietic stem cell transplantation may be a potential "cure" for refractory pemphigus.Therefore, this approach deserves further studies to evaluate the risk-benefit ratio in patients with pemphigus showing resistance to conventional therapy.

Key words: Pemphigus, Hematopoietic stem cells, Transplantation, autologous, Rituximab