Wiktor-Jedrzejczak W, Szczylik C, Pojda Z, Siekierzynski M, Kansy J, Klos M, Ratajczak M Z, Pejcz J, Jaskulski D, Gornas P
Eur J Haematol. 1987 Feb;38(2):204-6. doi: 10.1111/j.1600-0609.1987.tb01163.x.
A 6-yr-old girl with congenital corticosteroid-resistant pure red cell aplasia was treated with bone marrow transplant from her HLA-identical, MLC-unreactive sister in November 1984 following conditioning with busulfan and cyclophosphamide. Full engraftment was obtained and the patient at 21 months post-transplant is in excellent clinical condition maintaining normal red cell counts. We conclude that BMT should be considered as a therapy for at least the most severe cases of Diamond-Blackfan anaemia resistant to corticosteroids. Successful outcome of this therapy provides an argument for the stem cell origin of this disorder.
一名患有先天性皮质类固醇抵抗性纯红细胞再生障碍性贫血的6岁女孩，于1984年11月在接受白消安和环磷酰胺预处理后，接受了来自其HLA相同、混合淋巴细胞培养无反应的姐姐的骨髓移植。移植完全成功，移植后21个月的患者临床状况良好，红细胞计数维持正常。我们得出结论，骨髓移植应被视为至少对皮质类固醇耐药的最严重的先天性纯红细胞再生障碍性贫血病例的一种治疗方法。这种治疗方法的成功结果为这种疾病的干细胞起源提供了依据。
