Chung Nack-Gyun, Lee Jae Wook, Jang Pil-Sang, Jeong Dae-Chul, Cho Bin, Kim Hack-Ki
Division of Hematology and Oncology, Department of Pediatrics, The Catholic University of Korea, Seoul, Korea.
Pediatr Transplant. 2013 Jun;17(4):387-93. doi: 10.1111/petr.12073. Epub 2013 Apr 3.
We evaluated the results of a novel conditioning regimen of reduced dose cyclophosphamide (Cy, 25 mg/kg for four days), fludarabine (Flu, 30 mg/m(2) for four days), and rabbit ATG (2.5 mg/kg for three days) for allogeneic transplant of children with SAA, implemented since January 2009. Overall, 23 patients were treated with this regimen (16 male, seven female), including 10 diagnosed with VSAA. Donors included eight-MSD and 15 UD (five-matched UD, and 10 mismatched UD). All patients showed neutrophil and platelet engraftment. Cumulative incidence of acute (grade 2 or above) and chronic GVHD was 26.1% and 8.7%, respectively. Estimated two-yr FFS and OS for the entire cohort was 90.3 ± 6.5%. Rates of TRM and graft failure were 5.3% and 4.3%, respectively. No difference in OS was found according to disease severity (SAA vs. VSAA, p = 0.184), or according to donor type (MSD vs. UD, p = 0.699). Excellent outcomes of patients with VSAA underscore the efficacy of allogeneic transplant as a means of expediting hematopoietic recovery. Improved survival of UD transplant reaffirms its role as a valid therapeutic alternative in the absence of MSD.
我们评估了自2009年1月起实施的一种新型预处理方案的效果，该方案用于再生障碍性贫血（SAA）患儿的异基因移植，包括降低剂量的环磷酰胺（Cy，25mg/kg，共4天）、氟达拉滨（Flu，30mg/m²，共4天）和兔抗胸腺细胞球蛋白（2.5mg/kg，共3天）。总体而言，23例患者接受了该方案治疗（16例男性，7例女性），其中10例诊断为极重型再生障碍性贫血（VSAA）。供者包括8例无关供者（MSD）和15例单倍体相合供者（UD，5例全相合UD和10例不相合UD）。所有患者均实现中性粒细胞和血小板植入。急性（2级及以上）和慢性移植物抗宿主病（GVHD）的累积发生率分别为26.1%和8.7%。整个队列的估计2年无进展生存率（FFS）和总生存率（OS）为90.3±6.5%。移植相关死亡率（TRM）和移植物失败率分别为5.3%和4.3%。根据疾病严重程度（SAA与VSAA，p = 0.184）或供者类型（MSD与UD，p = 0.699），未发现OS有差异。VSAA患者的良好结局强调了异基因移植作为加速造血恢复手段的有效性。单倍体相合供者移植患者生存率的提高再次证实了其在无无关供者情况下作为有效治疗选择的作用。
