Abstract
The aim of this study is to explore the effectiveness 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 diagnosis of pemphigus was made on the basis of the clinical, histologic and immunofluorescence findings. The patient had shown resistance to conventional therapy with glucocorticoid 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 cyclophosphamide (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 transplantation was followed by reconstitution of the immune system which was monitored by flow cytometry. The glucocorticoid was withdrawn immediately after transplantation. The pemphigus titer turned negative 6 weeks after transplantation 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 peripheral hematopoietic stem cell transplantation may be a potential “cure” for refractory pemphigus. However, further studies are needed to evaluate the risk-benefit ratio of this approach in patients with pemphigus showing resistance to conventional therapy.
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References
Diao Y T, Zeng K, Sun J, Sun L D, Meng F Y, Zhou Z G, Liu Q F, Peng X B, Xu D, Huang L, Zhao J, Li J H. Autologous peripheral hematopoietic stem-cell transplantation in treatment with systemic lupus erythematosus. Zhonghua Pi Fu Ke Za Zhi, 2006, 39(6): 317–319 (in Chinese)
Passweg J, Tyndall A. Autologous stem cell transplantation in autoimmune diseases. Semin Hematol, 2007, 44(4): 278–285
Sibilia J. Treatment of systemic lupus erythematosus in 2006. Joint Bone Spine, 2006, 73(6): 591–598
Petri M, Brodsky R. High-dose cyclophosphamide and stem cell transplantation for refractory systemic lupus erythematosus. JAMA, 2006, 295(5): 559–560
Tyndall A, Daikeler T. Autologous hematopoietic stem cell transplantation for autoimmune diseases. Acta Haematol, 2005, 114(4): 239–247
Goldblatt F, Isenberg D A. New therapies for systemic lupus erythematosus. Clin Exp Immunol, 2005, 140(2): 205–212
Traynor A E, Barr W G, Rosa R M, Rodriguez J, Oyama Y, Baker S, Brush M, Burt R K. Hematopoietic stem cell transplantation for severe and refractoy lupus. Analysis after five years and fifteen patients. Arthitis Rheum, 2002, 46(11): 2917–2923
Jayne D. Stem cell transplantation in systemic lupus erythematosus. Best Pract Res Clin Haematol, 2004, 17(2): 291–304
España A, Fernández-Galar M, Lloret P, Sánchez-Ibarrola A, Panizo C. Long-term complete remission of sever penphigus vulgaris with monoclonal anti CD20 antibody therapy and immunophenotype correlations. J Am Acad Dermatol, 2004, 50(6): 974–976
Popat U, Krance R. Haematopoietic stem cell transplantation for autoimmune disorders: the American perspective. Br J Haematol, 2004, 126(5): 637–649
Musso M, Porretto F, Crescimanno A, Bondi F, Polizzi V, Scalone R. Intense immunosuppressive therapy followed by autologus peripheral blood selected progenitor cell reinfusion for severe autoimmune disease. Am J Hematol, 2001, 66(2): 75–79
Jayne D, Tyndall A. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus. 2004, 13(5): 359–365
Lisukov I A, Sizikova S A, Kulagin A D, Kruchkova I V, Gilevich A V, Konenkova L P, Zonova E V, Chernykh E R, Leplina O Y, Sentyakova T N, Demin A A, Kozlov V A. High-dose immunosuppression with autologous stem cell transplantation in severe refractory systemic lupus erythematosus. Lupus, 2004, 13(2): 89–94
Scheinberg P. Stem-cell transplantation for autoimmune diseases. Cytotherapy, 2003, 5(3): 243–251
Rosa S B, Voltarelli J C, Chies J A, Pranke P. The use of stem cells for the treatment of autoimmune diseases. Braz J Med Biol Res, 2007, 40(12): 1579–1597
Liu Q F, Sun J, Zhang Y, Xu D, Liu X L, Xu B, Meng F Y, Zhou S Y. Quadruple regimen cyclosporine A + methotrexate + mycophenolate mofetil + antithymosyte globulin in prevention of graft versus host disease in the transplantation of unrelated donor haematopoietic stem cell. Di Yi Jun Yi Da Xue Xue Bao, 2003, 23(11): 1143–1145 (in Chinese)
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Translated from Chinese Journal of Dermatology, 2007, 40(1): 13–15 [译自: 中华皮肤科杂志]
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Sun, L., Sun, J., Zeng, K. et al. Autologous peripheral hematopoietic stem-cell transplantation in a patient with refractory pemphigus. Front. Med. China 2, 191–194 (2008). https://doi.org/10.1007/s11684-008-0036-8
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DOI: https://doi.org/10.1007/s11684-008-0036-8