Allogeneic stem cell transplantation for children with acquired severe aplastic anaemia: a retrospective study by the Viva‐Asia Blood and Marrow Transplantation Group

Allogeneic stem cell transplantation for children with acquired severe aplastic anaemia: a retrospective study by the Viva‐Asia Blood and Marrow Transplantation Group
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DOI:
10.1111/bjh.12405
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发表时间:
2013-08
影响因子:
6.5
通讯作者:
Jing Chen;V. Lee;C. Luo;A. Chiang;S. Hongeng;P. Tan;A. Tan;Kleebsabai Sanpakit;Chun Fu Li
Jing Chen;V. Lee;C. Luo;A. Chiang;S. Hongeng;P. Tan;A. Tan;Kleebsabai Sanpakit;Chun Fu Li
中科院分区:
医学2区
文献类型:
--
作者:
Jing Chen;V. Lee;C. Luo;A. Chiang;S. Hongeng;P. Tan;A. Tan;Kleebsabai Sanpakit;Chun Fu Li

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我们回顾分析了在2000-2011年间在10个亚太机构中的一个机构接受造血干细胞移植(HSCT)的127例获得性重型再生障碍性贫血(SAA)儿童的结果。53例为相合同胞供者(MSD),74例为替代供者(AD),包括22例匹配的非亲缘供者、32例失配的非亲缘供者和20例失配的亲缘供者。平均随访45·5个月(13-139个月),与MSD组相比,AD患者有更多的II-IV级急性移植物抗宿主病(aGVHD;但III~IV级aGVHD(10.2%vs.12.5%,P=0.774)、移植物衰竭(15.1%vs.15.5%,P=0.658)和5年总存活率(90.6%vs.83.7%,P=0.251)相似。作为干细胞来源的外周血干细胞(PBSC)与骨髓(BM)相比,GF(18%vs.9.1%P=0.013)、II~IV级aGVHD(28.1%vs.17.4%,P=0.258)、慢性GVHD(25.8%vs.29.3%,P=0.822)和相似的结局(89.7%vs.82.4%,P=0.665)明显减少。在单因素分析中,GF(P<0·001)和II-IV级aGVHD(P=0.009)是预后不良的危险因素。在多因素分析中,仅有GF与生存不良有关(P=0.012)。在亚太地区,AD和PBSC HSCT的结果与MSD和BM HSCT相似。
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.