Salvage allogeneic hematopoietic SCT for primary graft failure in children
Salvage allogeneic hematopoietic SCT for primary graft failure in children
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DOI：
10.1038/bmt.2013.36
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发表时间：
2013-09-01
影响因子：
4.8
通讯作者：
Fukuda, T.
中科院分区：
文献类型：
--
作者：
Kato, M.;Matsumoto, K.;Fukuda, T.
原发性移植物衰竭（pGF）与相当大的发病率和死亡率相关。挽救性造血SCT（HSCT）可挽救pGF患者；然而，最佳的预处理方案和干细胞来源尚未确定，特别是对于儿童。在这项研究中，我们回顾性分析了 102 名接受 pGF 挽救性同种异体 HSCT 的儿科患者。与来自两种或三种抗原不匹配的相关供体 (rMM23) 或脐带血移植 (CBT) 的挽救性 HSCT 相比，来自匹配或一种抗原不匹配的相关供体 (rMM01) 的挽救性 HSCT 提供了更好的 OS。与 rMM23 相比，CBT 表现出着床率和晚着床成就略低的趋势；然而，两组之间的 OS 率相似（挽救 HSCT 后 1 年，rMM23 为 47.6+/-7.7%，CBT 为 45.7+/-8.6%）。多变量分析显示，使用氟达拉滨或放射治疗的预处理方案与较高的植入率相关，而使用烷化剂的预处理方案与更好的 OS 相关。总之，我们的结果表明，rMM01 是儿童 pGF 挽救性 HSCT 的最合适供体，对于没有 rMM01 的患者，与 rMM23 相比，CBT 是同样重要的选择。
Primary graft failure (pGF) is associated with considerable morbidity and mortality. Salvage hematopoietic SCT (HSCT) can rescue pGF patients; however, the optimal preconditioning regimen and stem cell source are yet to be determined, particularly in children. In this study, we retrospectively analyzed 102 pediatric patients who received salvage allogeneic HSCT for pGF. Salvage HSCT from matched or one-Ag-mismatched related donors (rMM01) provided superior OS compared with that from two- or three-Ags-mismatched related donors (rMM23) or cord blood transplantation (CBT). CBT showed a trend toward a slightly lower engraftment rate and late engraftment achievement compared with rMM23; however, the OS rate was similar between the two groups (47.6+/-7.7% for rMM23 and 45.7+/-8.6% for CBT, at 1 year after salvage HSCT). Multivariate analysis showed that preconditioning regimens with fludarabine or irradiation were associated with a higher engraftment rate and those with alkylating agents were associated with better OS. In conclusion, our results showed that rMM01 was the most suitable donor for salvage HSCT for pediatric pGF, and that CBT was an equally important option compared with rMM23 for patients without rMM01.