A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia
A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia
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DOI：
10.1038/bmt.2012.79
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发表时间：
2012-12-01
影响因子：
4.8
通讯作者：
Huang, X. J.
中科院分区：
文献类型：
--
作者：
Xu, L. P.;Liu, K. Y.;Huang, X. J.
严重再生障碍性贫血（SAA）的造血干细胞移植（HSCT）的不匹配的相关供者面临的挑战主要与移植失败和GVHD相关。本文报告了本中心连续19例半相合家系造血干细胞移植的SAA/very SAA（VSAA）患者，其中18例有2-3个位点错配。所有19例患者均对既往治疗无效，并在移植前大量输血。HSCT前预处理方案包括BU、CY和胸腺球蛋白。接受CsA、霉酚酸酯（MMF）和短期MTX预防GVHD。干细胞移植物的来源是G-CSF致敏的BM和G-CSF动员的外周血干细胞的组合。所有患者均实现了100%的供体骨髓移植;骨髓移植的中位时间为12天（范围为10-29天），血小板移植的中位时间为18天（范围为8-180天），累积血小板移植发生率为84.21 +/-10.53%。II-IV级急性GVHD的累积发生率为42.1 +/- 11.3%，慢性GVHD为56.2 +/- 12.4%。生存患者的OS为64.6 +/- 12.4%，中位随访时间为746天（90-1970）。这些有限的回顾性分析数据表明，HLA半相合HSCT SAA患者没有HLA相合同胞供体可能是可行的。未来需要进一步研究通过减少GVHD来增加OS，同时保持稳定的植入。骨髓移植（2012）47，1507-1512; doi：10.1038/bmt.2012.79; 2012年5月28日在线发表
Mismatched related donors of hematopoietic SCT (HSCT) for severe aplastic anemia (SAA) present challenges mainly associated with graft failure and GVHD. The greater the HLA disparity, the poorer the OS. About 19 consecutive SAA/very SAA (VSAA) patients who received HSCT from haploidentical family donors in our center are reported in this study, 18/19 pairs had 2-3 loci mismatched. All 19 cases failed to respond to previous therapy and were heavily transfused before transplantation. The conditioning regimen before HSCT included BU, CY and thymoglobulin. The recipients received CsA, mycophenolate mofetil (MMF) and short-term MTX for GVHD prophylaxis. The source of stem cell grafts was a combination of G-CSF-primed BM and G-CSF-mobilized peripheral blood stem cells. All patients achieved 100% donor myeloid engraftment; the median time for myeloid engraftment was 12 days (ranging from 10-29 days) and for platelets was 18 days (ranging from 8-180 days) with a cumulative platelet engraftment incidence of 84.21 +/- 10.53%. The cumulative incidence was 42.1 +/- 11.3% for grade II-IV acute GVHD and 56.2 +/- 12.4% for chronic GVHD. The OS was 64.6 +/- 12.4% with a median 746-day (90-1970) follow-up for surviving patients. These limited retrospective analysis data suggest that HLA-haploidentical HSCT for SAA patients without an HLA-identical sibling donor might be feasible. Further research to increase OS by decreasing GVHD while maintaining stable engraftment will be needed in the future. Bone Marrow Transplantation (2012) 47, 1507-1512; doi:10.1038/bmt.2012.79; published online 28 May 2012