Brochstein J A, Kirkpatrick D, Giardina P J, Weinberg R S, Alter B P, Driscoll C, Wolfe L, Shank B, O'Reilly R J
Br J Haematol. 1986 Jul;63(3):445-56. doi: 10.1111/j.1365-2141.1986.tb07521.x.
Bone marrow transplantation has generally been unsuccessful when applied to patients with thalassaemia major over the age of 6 years. We report here two successful transplants for this disorder in a 7 1/2-year-old boy and an 11-year-old girl following a pre-transplant cytoreductive/immunosuppressive regimen of total body irradiation and cyclophosphamide. Complete durable engraftment of donor haematopoietic and lymphoid populations was documented through several approaches, including cytogenetic analysis, haemoglobin electrophoresis, globin chain synthetic ratios, red cell typing and DNA restriction enzyme analysis. Both patients are surviving in good health, 28 and 9 months from transplantation. The successful outcome in these patients demonstrates the feasibility of marrow transplantation for the treatment of thalassaemia in multiply transfused and, presumably, highly sensitized patients.
对于6岁以上的重型地中海贫血患者，骨髓移植通常不成功。我们在此报告，一名7岁半男孩和一名11岁女孩在接受全身照射和环磷酰胺的移植前细胞减灭/免疫抑制方案后，成功进行了针对这种疾病的两次移植。通过几种方法记录了供体造血和淋巴群体的完全持久植入，包括细胞遗传学分析、血红蛋白电泳、珠蛋白链合成比率、红细胞分型和DNA限制性内切酶分析。两名患者移植后分别存活了28个月和9个月，身体健康。这些患者的成功结果表明，对于多次输血且可能高度致敏的患者，骨髓移植治疗地中海贫血是可行的。
