Bordigoni P
Ann Pediatr (Paris). 1989 Oct;36(8):545-55.
Malignant hemopathies and immune deficiencies are the main indications for allogeneic bone marrow transplantation in children. Among the former, the most common condition is acute lymphoblastic leukemia, in which a bone marrow transplant can be performed during the second or first complete remission (CR). Thirty to 50% and 60 to 75% of these grafts, respectively, are successful. The success rate is 50 to 70% among patients with acute myeloblastic leukemia grafted during the first complete remission, and among patients with chronic myeloid leukemia grafted during the chronic phase. Severe medullary aplasia and Fanconi disease are undoubtedly good indications for bone marrow transplantation, which has a 60 to 70% success rate. Severe combined immune deficiencies (SCID) and Wiskott-Aldrich disease are also good indications for HLA-identical bone marrow transplantation, which is successful in 60% of cases. Among the metabolic diseases, good results have been obtained only in Hurler disease and Gaucher disease. Questionable indications include thalassemia, Blackfan-Diamond disease, and chronic granulomatous disease. Results are disappointing in most metabolic diseases, as well as in non-HLA-identical transplantations in diseases other than SCID.
恶性血液病和免疫缺陷是儿童同种异体骨髓移植的主要适应证。在前者中，最常见的疾病是急性淋巴细胞白血病，可在第二次或第一次完全缓解期（CR）进行骨髓移植。这些移植分别有30%至50%和60%至75%获得成功。在第一次完全缓解期进行移植的急性髓细胞白血病患者以及在慢性期进行移植的慢性髓性白血病患者中，成功率为50%至70%。严重骨髓发育不全和范可尼贫血无疑是骨髓移植的良好适应证，成功率为60%至70%。严重联合免疫缺陷（SCID）和威斯科特-奥尔德里奇综合征也是HLA匹配的骨髓移植的良好适应证，60%的病例获得成功。在代谢性疾病中，仅在黏多糖贮积症Ⅰ型和戈谢病中取得了良好效果。存在疑问的适应证包括地中海贫血、先天性纯红细胞再生障碍性贫血和慢性肉芽肿病。在大多数代谢性疾病以及SCID以外疾病的非HLA匹配移植中，结果令人失望。
