Umbilical-cord blood cell transplantation conditioned with a reduced intensity-regimen is a practical salvage therapy for severe aplastic anemia refractory to immunosuppressive therapy with antithymocyte globulin/ciclosporin.
Immunosuppressive therapy and stem cell transplantation from an HLA-identical donor are the major effective treatments for severe aplastic anemia. However, treatments still need to be developed for patients who do not have a HLA-identical donor and have not shown a clinical response to immunosuppressive therapy. We herein report on 2 patients in whom this problem could be overcome by transplantation of HLA-mismatched umbilical cord blood from unrelated donors. Two Japanese patients with severe aplastic anemia underwent conditioning with fludarabine, cyclophosphamide, and low-dose total-body irradiation and then received transplants of umbilical cord blood. Engraftment of the three lineages occurred without problems. We conclude that umbilical cord blood transplantation with a reduced-intensity conditioning regimen of fludarabine, cyclophosphamide, and total-body irradiation for patients with aplastic anemia is a practical treatment and may be an attractive alternative for patients who does not have an HLA-identical donor and have shown no clinical response to immunosuppressive therapy.
Tajika
Kenji
K
Department of Pathophysiological Management/Medical Oncology, Graduate School of Medicine, Nippon Medical School, Tokyo, Japan. tajika@nms.ac.jp
Mizuki
Taro
T
Nakayama
Kazutaka
K
Yamaguchi
Hiroki
H
Dan
Kazuo
K
D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D016572:Cyclosporine / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D008297:Male; D016879:Salvage Therapy; D019172:Transplantation Conditioning / Q000379:methods*
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine