Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34+ cells and a fixed CD3+ dose
Haploidentical related-donor hematopoietic cell transplantation in children using megadoses of CliniMACs-selected CD34+ cells and a fixed CD3+ dose
复制标题
DOI：
10.1038/bmt.2012.186
复制
发表时间：
2013-04-01
影响因子：
4.8
通讯作者：
Cowan, M. J.
中科院分区：
文献类型：
--
作者：
Dvorak, C. C.;Gilman, A. L.;Cowan, M. J.
我们利用 CliniMACs 系统进行了一项前瞻性 II 期试验，对来自半相合供体的 PBSC 进行 CD34(+) 细胞选择，以评估植入和血液免疫重建。总共有 21 名患有血液恶性肿瘤或非恶性疾病的儿童接受了 1200 cGy TBI、塞替派、氟达拉滨和胸腺球蛋白的调理。患者接受大剂量的CD34(+)细胞（中位值：22 x 10(6)/kg），固定剂量为3 x 10(4)/kg CD3(+)细胞/kg，移植发生率为90%，中性粒细胞和血小板迅速恢复。 32%（95%置信区间（CI），15-54%）的可评估患者出现II级急性GVHD（aGVHD），没有出现III-IV级aGVHD，35%（95%CI，17-59%）的患者出现慢性广泛GVHD。预计 2 年 EFS 为 62%（95% CI，48-83%），中位幸存者随访时间为 49 个月（范围：18-119 个月）。非恶性疾病患者的 2 年 EFS 估计为 100%（95% CI，56-100%），缓解期恶性肿瘤患者的 2 年 EFS 估计为 56%（95% CI，22-89%）。来自半相合相关供体的大剂量CD34(+)细胞和固定CD3(+)细胞剂量为患有非恶性疾病的儿科患者和缓解期移植的恶性疾病患者带来了良好的结果。骨髓移植 (2013) 48, 508-513; doi：10.1038/bmt.2012.186； 2012 年 11 月 26 日在线发布
We conducted a prospective phase II trial utilizing the CliniMACs system to perform CD34(+)-cell selection of PBSCs from haploidentical donors to evaluate engraftment and hematoimmunological reconstitution. In total, 21 children with hematological malignancies or nonmalignant conditions underwent conditioning with 1200 cGy TBI, thiotepa, fludarabine and Thymoglobulin. Patients received megadoses of CD34(+) cells (median: 22 x 10(6)/kg) with a fixed dose of 3 x 10(4)/kg CD3(+) cells/kg, and engraftment occurred in 90% with prompt recovery of neutrophils and platelets. Grade II acute GVHD (aGVHD) was seen in 32% (95% confidence interval (CI), 15-54%) of evaluable patients, there was no grade III-IV aGVHD, and chronic extensive GVHD was seen in 35% (95% CI, 17-59%) of patients. The estimated 2-year EFS was 62% (95% CI, 48-83%) with a median survivor follow-up of 49 months (range: 18-119 months). Patients with nonmalignant diseases had an estimated 2-year EFS of 100% (95% CI, 56-100%) and patients with malignancies in remission had an estimated 2-year EFS of 56% (95% CI, 22-89%). Megadose CD34(+) cells with a fixed CD3(+) cell dose from haploidentical related donors resulted in good outcomes for pediatric patients with nonmalignant diseases and those with malignant diseases transplanted in remission. Bone Marrow Transplantation (2013) 48, 508-513; doi: 10.1038/bmt.2012.186; published online 26 November 2012