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首页> 外文期刊>British Journal of Haematology >Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: A United Kingdom multicentre retrospective experience
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Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: A United Kingdom multicentre retrospective experience

机译：免疫抑制治疗失败后的小儿再生障碍性贫血中匹配的无关供体移植的出色结果：英国多中心回顾性经验

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摘要

We retrospectively analysed the outcome of consecutive children with idiopathic severe aplastic anaemia in the United Kingdom who received immunosuppressive therapy (IST) or matched unrelated donor (MUD) haematopoietic stem cell transplantation (HSCT). The 6-month cumulative response rate following rabbit antithymocyte globulin (ATG)/ciclosporin (IST) was 32·5% (95% CI 19·3-46·6) (n = 43). The 5-year estimated failure-free survival (FFS) following IST was 13·3% (95% confidence interval [CI] 4·0-27·8). In contrast, in 44 successive children who received a 10-antigen (HLA-A, -B, -C, -DRB1, -DQB1) MUD HSCT there was an excellent estimated 5-year FFS of 95·01% (95% CI 81·38-98·74). Forty of these children had failed IST previously. HSCT conditioning was a fludarabine, cyclophosphamide and alemtuzumab (FCC) regimen and did not include radiotherapy. There were no cases of graft failure. Median donor chimerism was 100% (range 88-100%). A conditioning regimen, such as FCC that avoids total body irradiation is ideally suited in children. Our data suggest that MUD HSCT following IST failure offers an excellent outcome and furthermore, if a suitable MUD can be found quickly, MUD HSCT may be a reasonable alternative to IST.
机译：我们回顾性分析了在英国接受免疫抑制疗法（IST）或相配的无关供体（MUD）造血干细胞移植（HSCT）的连续性特发性再生障碍性贫血儿童的结局。兔抗胸腺细胞球蛋白（ATG）/环孢菌素（IST）给药后6个月累积应答率为32·5％（95％CI 19·3-46·6）（n = 43）。 IST后的5年估计无故障生存期（FFS）为13·3％（95％置信区间[CI] 4·0-27·8）。相反，在44位连续接受10抗原（HLA-A，-B，-C，-DRB1，-DQB1）MUD HSCT的儿童中，其5年FFS估计为95·01％（95％CI 81·38-98·74）。这些孩子中有40个以前的IST失败。 HSCT条件为氟达拉滨，环磷酰胺和阿仑单抗（FCC）方案，不包括放射疗法。没有移植失败的案例。中位供体嵌合率为100％（范围88-100％）。避免全身照射的条件疗法（例如FCC）非常适合儿童。我们的数据表明，IST失败后的MUD HSCT提供了出色的结果，此外，如果能够快速找到合适的MUD，MUD HSCT可能是IST的合理替代方案。

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