Müller B U, Tichelli A, Passweg J R, Nissen C, Wodnar-Filipowicz A, Gratwohl A
Department of Medicine, Kantonsspital, University of Basel, Switzerland.
Bone Marrow Transplant. 1999 Jun;23(11):1205-7. doi: 10.1038/sj.bmt.1701785.
This case describes a 16-year-old woman treated successfully by a bone marrow transplant from her HLA-identical brother for refractory acquired pure red cell aplasia. Conditioning was as for severe aplastic anaemia with cyclophosphamide 4 x 50 mg/kg and antithymocyte globulin. Complete donor type engraftment at 3 months reversed to full autologous reconstitution at 2 years with normal haemopoiesis. The potential implications on pathogenesis of the disease as well as on treatment of autoimmune disorders by stem cell transplantation are discussed.
该病例描述了一名16岁女性,她因难治性获得性纯红细胞再生障碍性贫血接受了来自其 HLA 相同的哥哥的骨髓移植,并获得成功。预处理方案与重型再生障碍性贫血相同,使用环磷酰胺4×50mg/kg及抗胸腺细胞球蛋白。3个月时完全为供体类型的植入,2年时转变为完全自体造血重建,造血功能正常。本文讨论了该疾病发病机制以及干细胞移植治疗自身免疫性疾病的潜在意义。