Department of Haematology/Oncology, Shanghai Jiao Tong University School of Medicine, Shanghai Children's Medical Centre, Shanghai, China.
Br J Haematol. 2013 Aug;162(3):383-91. doi: 10.1111/bjh.12405. Epub 2013 Jun 5.
We retrospectively analysed the outcomes of 127 children with acquired severe aplastic anaemia (SAA) who had received haematopoietic stem cell transplantation (HSCT) between 2000 and 2011 in one of the 10 Asia Pacific institutions. Fifty-three were matched sibling donor (MSD) and 74 were alternative donor (AD), including 22 matched unrelated donor, 32 mismatched unrelated donor and 20 mismatched related donor. With a median follow up 45.5 months (13-139) and when compared to the MSD group, AD recipients had more grade II-IV acute graft-versus-host disease (aGVHD; 14.3% vs. 32.8%, P = 0.029), but similar grade III-IV aGVHD (10.2% vs. 12.5%, P = 0.774), graft failure (GF) (15.1% vs. 15.5%, P = 0.658) and 5-year overall survival (90.6% vs. 83.7%, P = 0.251). As a source of stem cell, peripheral blood stem cells (PBSC) resulted in less GF (18% vs. 9.1% P = 0.013), similar grade II-IV aGVHD (28.1% vs. 17.4%, P = 0.258), chronic GVHD (25.8% vs. 29.3%, P = 0.822) and similar outcomes (89.7% vs. 82.4%, P =0.665) when compared to bone marrow (BM). In univariate analysis, GF (P < 0.001) and grade II-IV aGVHD (P = 0.009) were predictors of poor survival. In multivariate analysis, only GF was associated with poor survival (P = 0.012). The outcome of AD and PBSC HSCT were comparable to that of MSD and BM HSCT in the Asia Pacific region.
我们回顾性分析了 2000 年至 2011 年间亚太地区 10 家机构 127 例获得性严重再生障碍性贫血（SAA）患儿接受造血干细胞移植（HSCT）的结果。53 例为同胞供者（MSD），74 例为非亲缘供者（AD），包括 22 例匹配的无关供者、32 例不匹配的无关供者和 20 例不匹配的亲缘供者。中位随访时间为 45.5 个月（13-139），与 MSD 组相比，AD 组患者Ⅱ-Ⅳ级急性移植物抗宿主病（aGVHD）发生率更高（14.3%比 32.8%，P = 0.029），但Ⅲ-Ⅳ级 aGVHD 发生率相近（10.2%比 12.5%，P = 0.774）、移植物失败（GF）（15.1%比 15.5%，P = 0.658）和 5 年总生存率（90.6%比 83.7%，P = 0.251）。作为干细胞来源，外周血干细胞（PBSC）导致 GF 发生率更低（18%比 9.1%，P = 0.013）、Ⅱ-Ⅳ级 aGVHD 发生率相近（28.1%比 17.4%，P = 0.258）、慢性移植物抗宿主病（CGVHD）发生率相近（25.8%比 29.3%，P = 0.822），结局相似（89.7%比 82.4%，P =0.665）。与骨髓（BM）相比。单因素分析显示，GF（P < 0.001）和Ⅱ-Ⅳ级 aGVHD（P = 0.009）是生存不良的预测因素。多因素分析显示，只有 GF 与不良生存相关（P = 0.012）。在亚太地区，AD 和 PBSC HSCT 的结果与 MSD 和 BM HSCT 相当。
