Department of Hematology, People's Hospital and Institute of Hematology, Peking University, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, PR China.
Bone Marrow Transplant. 2012 Dec;47(12):1507-12. doi: 10.1038/bmt.2012.79. Epub 2012 May 28.
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.
在重型再生障碍性贫血（SAA）患者中，造血干细胞移植（HSCT）的供者不合主要与移植物衰竭和移植物抗宿主病（GVHD）有关。HLA 错配程度越高，总生存率（OS）越低。本研究报道了我院采用单倍体相合亲缘供者行 HSCT 的 19 例连续 SAA/极重型 SAA（VSAA）患者，18/19 对存在 2-3 个 HLA 位点错配。所有患者在移植前均未对先前的治疗产生反应，且需要大量输血。HSCT 前的预处理方案包括白消安（BU）、环磷酰胺（CY）和胸腺球蛋白。受者接受环孢素 A（CsA）、霉酚酸酯（MMF）和短程甲氨蝶呤（MTX）预防 GVHD。干细胞移植物的来源是 G-CSF 预激骨髓和 G-CSF 动员外周血干细胞的组合。所有患者均达到 100%供者髓系嵌合；中性粒细胞植入的中位时间为 12 天（范围 10-29 天），血小板植入的中位时间为 18 天（范围 8-180 天），血小板植入累积发生率为 84.21±10.53%。Ⅱ-Ⅳ级急性 GVHD 的累积发生率为 42.1±11.3%，慢性 GVHD 的累积发生率为 56.2±12.4%。OS 为 64.6±12.4%，生存患者的中位随访时间为 746 天（90-1970 天）。这些有限的回顾性分析数据表明，对于没有 HLA 完全相合同胞供者的 SAA 患者，HLA 单倍体相合 HSCT 可能是可行的。未来需要进一步研究，通过降低 GVHD 发生率同时保持稳定的嵌合状态来提高 OS。
