Hutspardol Sakara, Sirachainan Nongnuch, Anurathapan Usanarat, Pakakasama Samart, Songdej Duantida, Chuansumrit Ampaiwan, Sirireung Somtawin, Panthangkool Wanpen, Hongeng Suradej
Division ofHematology-Oncology, Department ofPediatrics, Faculty ofMedicine, Srinakharinwirot University Nakhon Nayok, Thailand.
J Med Assoc Thai. 2013 Jan;96 Suppl 1:S18-24.
Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a potentially curative treatment for severe aplastic anemia (SAA). This is a single institutional review to study the feasibility of using allo-SCT for Thai children with SAA.
Nine children with SAA (7 matched-sibling donor-SCT, 1 matched-unrelated donor-SCT and 1 haploidentical-SCT) underwent allo-SCT between October 2002 and September 2010. Cyclophosphamide and anti-thymocyte globulin (CY/ATG) were used as conditioning regimen for 4 patients with matched-sibling donor-SCT CY/ATG and fludarabine were used for 3 patients with matched-sibling donor-SCT and one patient with haplo-identical SCT. One matched-unrelated donor-SCT received CY/ATG and total body irradiation.
Eight of 9 patients (89%) achieved neutrophil engraftment within 13.5 days (range 6.0-22.0). One matched-sibling donor-SCT recipient who failed to achieve engraftment died from acute renal failure and gram-negative sepsis on day 21 post allo-SCT. One matched-sibling donor-SCT case developed late graft failure on day 72 and died from invasive fungal infection. For graft versus host disease (GVHD), a haplo-identical-SCT patient died from steroid refractory grade IV acute GVHD. At last follow-up, six patients (67%) alive at a median follow-up time of 76.4 months (range 2.3-88.8). Overall survival (OS) and event-free survival (EFS) at 5 year was 63% and 65%, respectively.
Allo-SCT is a feasible curative treatment for children with SAA in Thailand. Graft failure and severe GVHD in alternative donors SCT are responsible for major causes of death. OS and EFS probabilities are stable after the first year post transplant.
异基因造血干细胞移植（allo-SCT）是治疗重型再生障碍性贫血（SAA）的一种潜在治愈性疗法。这是一项单机构研究，旨在探讨allo-SCT用于泰国SAA患儿的可行性。
2002年10月至2010年9月期间，9例SAA患儿（7例同胞全相合供者-SCT、1例无关全相合供者-SCT和1例单倍体相合-SCT）接受了allo-SCT。4例同胞全相合供者-SCT患者采用环磷酰胺和抗胸腺细胞球蛋白（CY/ATG）作为预处理方案；3例同胞全相合供者-SCT患者和1例单倍体相合SCT患者采用CY/ATG和氟达拉滨。1例无关全相合供者-SCT患者接受CY/ATG和全身照射。
9例患者中有8例（89%）在13.5天内（范围6.0 - 22.0天）实现中性粒细胞植入。1例同胞全相合供者-SCT受者未实现植入，在allo-SCT后第21天死于急性肾衰竭和革兰阴性菌败血症。1例同胞全相合供者-SCT病例在第72天发生晚期移植物失败，死于侵袭性真菌感染。对于移植物抗宿主病（GVHD），1例单倍体相合-SCT患者死于类固醇难治性IV级急性GVHD。在最后一次随访时，6例患者（67%）存活，中位随访时间为76.4个月（范围2.3 - 88.8个月）。5年总生存率（OS）和无事件生存率（EFS）分别为63%和65%。
allo-SCT是泰国SAA患儿可行的治愈性治疗方法。替代供者SCT中的移植物失败和严重GVHD是主要死亡原因。移植后第一年之后，OS和EFS概率稳定。
