Abstract
HLA-haploidentical hematopoietic stem cell transplantation (haplo-SCT) in HLA-homozygous patients is accompanied by HLA mismatches only in the host-versus-graft vector, and thus theoretically could be performed with standard graft-versus-host disease (GVHD) prophylaxis. However, the risk of GVHD remains uncertain, and graft failure could be a problem. In this study, we assessed nine HLA-homozygous patients who underwent haplo-SCT. Preparative treatment was cyclophosphamide/total body irradiation-based regimen in five patients, fludarabine/busulfan-based regimen in two, and other regimens in two. GVHD prophylaxis consisted of cyclosporine and methotrexate in seven patients, cyclosporine and mycophenolate mofetil in one, and cyclosporine alone in one. Seven patients achieved neutrophil engraftment and platelet recovery. The median times to neutrophil engraftment and platelet recovery were 15 and 44 days, respectively. Two patients developed graft failure, including one who achieved engraftment with a second SCT from the same donor. Grade II GVHD was observed in half of the evaluable patients; grades III and IV were not observed. Two patients died from treatment-related causes. Five patients were alive after a median follow-up period of 563 days. The probability of overall survival at 5 years was 65 %. These findings may serve as a rationale for considering haplo-SCT as a treatment option for HLA-homozygous patients.
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Acknowledgments
We thank the medical, nursing, and laboratory staff of the participating departments for their contributions to this study. We are also grateful to Ms. Aya Yano, Ms. Kimiko Yamamoto, and Mr. Koji Hayashi for their technical assistance and to Ms. Saori Hatemura, Ms. Kazuko Saida, and Ms. Kumiko Sugawara for their assistance with data collection. This study was supported in part by a grant from the Japanese Ministry of Health, Labor, and Welfare.
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Ikegame, K., Kaida, K., Yoshihara, S. et al. Feasibility of unmanipulated haploidentical stem cell transplantation using standard GVHD prophylaxis for HLA-homozygous patients. Int J Hematol 96, 101–108 (2012). https://doi.org/10.1007/s12185-012-1097-7
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DOI: https://doi.org/10.1007/s12185-012-1097-7