Tajika Kenji, Mizuki Taro, Nakayama Kazutaka, Yamaguchi Hiroki, Dan Kazuo
Department of Pathophysiological Management/Medical Oncology, Graduate School of Medicine, Nippon Medical School, Tokyo, Japan.
J Nippon Med Sch. 2007 Dec;74(6):424-9. doi: 10.1272/jnms.74.424.
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.
免疫抑制疗法和来自 HLA 匹配供体的干细胞移植是重型再生障碍性贫血的主要有效治疗方法。然而，对于没有 HLA 匹配供体且对免疫抑制疗法无临床反应的患者，仍需开发新的治疗方法。我们在此报告 2 例患者，通过移植来自无关供体的 HLA 不匹配脐带血克服了这一问题。两名日本重型再生障碍性贫血患者接受了氟达拉滨、环磷酰胺和低剂量全身照射的预处理，然后接受了脐带血移植。三个谱系的植入均未出现问题。我们得出结论，对于再生障碍性贫血患者，采用氟达拉滨、环磷酰胺和全身照射的低强度预处理方案进行脐带血移植是一种切实可行的治疗方法，对于没有 HLA 匹配供体且对免疫抑制疗法无临床反应的患者可能是一种有吸引力的替代方案。
