Excellent Outcome of Haploidentical Hematopoietic Stem Cell Transplantation in Children and Adolescents with Acquired Severe Aplastic Anemia
Excellent Outcome of Haploidentical Hematopoietic Stem Cell Transplantation in Children and Adolescents with Acquired Severe Aplastic Anemia
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DOI：
10.1016/j.bbmt.2013.01.023
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发表时间：
2013-05-01
影响因子：
4.3
通讯作者：
Seo, Jong Jin
中科院分区：
文献类型：
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作者：
Im, Ho Joon;Koh, Kyung Nam;Seo, Jong Jin
我们评估了患有获得性重型再生障碍性贫血（SAA）的儿童和青少年接受体外去除T细胞的外周血干细胞进行单倍体相合造血干细胞移植（HHCT）的结局。2009年7月至2012年7月期间，12例获得性SAA患者共接受了15例体外CD 3去除移植物的HHCT。在12例患者中，11例在HHCT后中位10天（范围，9 - 13天）实现中性粒细胞植入。1例患者未能实现一期植入，2例在植入后不久发生移植物排斥反应。所有3例发生早期移植物衰竭的患者均接受了第二次HHCT，并实现了持续植入。因此，最终植入率为100%。在9例患者中评估了急性移植物抗宿主病，不包括3例早期移植物衰竭患者。其中3例患者发生急性移植物抗宿主病（2例≥ II级，1例III级）。所有12例患者均存活，中位随访时间为14.3个月（范围：4.1至40.7个月），不依赖输血。造血干细胞移植从单倍体相合的家庭供者在体外CD 3 T细胞耗竭是一个合理的治疗选择，儿童和青少年获得性SAA。我们未来的试验将有助于解决与HHCT相关的问题，并为HHCT作为SAA治疗的进一步发展提供有价值的平台。(C)2013年美国血液和骨髓移植协会。
We evaluated the outcome of children and adolescents with acquired severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (HHCT) with in vitro T cell depleted peripheral blood stem cells. Twelve patients with acquired SAA received a total of 15 HHCTs with in vitro CD3-depleted grafts between July 2009 and July 2012. Among the 12 patients, 11 achieved neutrophil engraftment at a median of 10 days (range, 9 to 13 days) after HHCT. One patient failed to achieve primary engraftment, and two experienced graft rejection soon after engraftment. All three patients who experienced early graft failure received a second HHCT and achieved sustained engraftment. Thus, the final engraftment rate was 100%. Acute graft-versus-host disease was assessed in 9 patients, excluding the 3 patients with early graft failure. Three of these patients developed acute graft-versus-host disease (two >= grade II and one with grade III). All 12 patients survived and were transfusion-independent at a median follow-up of 14.3 months (range, 4.1 to 40.7 months). Hematopoietic stem cell transplantation from haploidentical family donors with in vitro CD3 T cell depletion is a reasonable therapeutic option for children and adolescents with acquired SAA. Our future trial with a uniform protocol will help to solve the problems associated with HHCT and provide a valuable platform for the further development of HHCT as a therapy for SAA. (C) 2013 American Society for Blood and Marrow Transplantation.