Margolis D, Camitta B, Pietryga D, Keever-Taylor C, Baxter-Lowe L A, Pierce K, Kupst M J, French J, Truitt R, Lawton C, Murray K, Garbrecht F, Flomenberg N, Casper J
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, USA.
Br J Haematol. 1996 Jul;94(1):65-72. doi: 10.1046/j.1365-2141.1996.d01-1772.x.
Alternative donor bone marrow transplantation (BMT) to treat severe aplastic anaemia (SAA) in children and young adults has been complicated by high rates of graft rejection and severe graft-versus-host disease (GVHD). We hypothesized that increased immunosuppression combined with T-cell depletion of the marrow graft would enable successful use of unrelated donor BMT in this disease. Preconditioning consisted of cytosine arabinoside, cyclophosphamide, and total body irradiation (TBI). T-cell depletion was with the anti-CD3 antibody T10B9. GVHD prophylaxis consisted of cyclosporine A. 28 previously transfused patients were transplanted. Nine donor/recipient pairs were HLA matched. As of 1 January 1996, 15/28 (54%) patients are alive, transfusion independent and well with a range of follow-up of 13 months to 8 years (median 2.75 years). Fatalities include all three patients with nonengraftment and all three patients with grade III/IV GVHD. Other fatalities were due to infections or therapy-related toxicity. The incidence >or= grade II acute GVHD was 28%. These data show that in children with SAA who have failed immunosuppression, unrelated donor BMT offers a reasonable hope of long-term survival.
采用替代供体骨髓移植（BMT）治疗儿童和青年重症再生障碍性贫血（SAA）时，出现了较高的移植物排斥率和严重的移植物抗宿主病（GVHD）等并发症。我们推测，增加免疫抑制并联合对骨髓移植物进行T细胞去除，将能够成功地在这种疾病中使用无关供体BMT。预处理方案包括阿糖胞苷、环磷酰胺和全身照射（TBI）。使用抗CD3抗体T10B9进行T细胞去除。采用环孢素A预防GVHD。对28例先前接受过输血的患者进行了移植。9对供体/受体在HLA上相匹配。截至1996年1月1日，28例患者中有15例（54%）存活，无需输血且状况良好，随访时间为13个月至8年（中位时间为2.75年）。死亡病例包括所有3例未植入的患者和所有3例发生III/IV级GVHD的患者。其他死亡原因是感染或与治疗相关的毒性反应。II级及以上急性GVHD的发生率为28%。这些数据表明，对于免疫抑制治疗失败的SAA儿童，无关供体BMT提供了长期存活的合理希望。
