Watanabe Nobuhiro, Takahashi Yoshiyuki, Matsumoto Kimikazu, Horikoshi Yasuo, Hama Asahito, Muramatsu Hideki, Yoshida Nao, Yagasaki Hiroshi, Kudo Kazuko, Horibe Keizo, Kato Koji, Kojima Seiji
Children's Medical Center, Japanese Red Cross, Nagoya, Japan.
Pediatr Transplant. 2011 Sep;15(6):642-9. doi: 10.1111/j.1399-3046.2011.01544.x. Epub 2011 Jul 15.
Although some studies have reported that TBI and MEL offer an effective conditioning regimen for autologous SCT in acute leukemia, little has been reported regarding outcomes of allogeneic SCT. We retrospectively evaluated outcomes for 50 pediatric patients who underwent allo-SCT conditioned with intravenous MEL (180-210 mg/m(2) ) and fractionated TBI (12-13.2 Gy) from HLA-identical related donors. Nineteen patients were in CR1, 18 were in CR2, and 13 showed advanced-stage disease (≥ CR3). Patients had received allo-SCT from HLA-identical siblings (n = 45) or phenotypically HLA-identical family donors (n = 5). Median duration of follow-up for all disease-free patients was 61 months (range, 8.8-177 months). At the time of analysis, 12 patients had died. Eleven of those died of relapse, and one died of TRM. DFS rates for all patients, patients with AML (n = 12), and patients with lymphoid malignancy (n = 38) were 61.4% and 82.1%, respectively. DFS rates for CR1, CR2, and ≥CR3 cases were 89.2%, 88.1%, and 23.1%, respectively (p < 0.05). MEL/TBI for pediatric patients with hematological malignancies was associated with lower relapse rates and no increase in toxicity, resulting in better survival.
尽管一些研究报告称，对于急性白血病患者的自体造血干细胞移植（SCT）而言，颅脑创伤（TBI）和马法兰（MEL）提供了一种有效的预处理方案，但关于异基因SCT结果的报道却很少。我们回顾性评估了50例接受异基因SCT的儿科患者的结果，这些患者接受了来自 HLA 相同的相关供体的静脉注射 MEL（180 - 210 mg/m²）和分次TBI（12 - 13.2 Gy）预处理。19例患者处于完全缓解期1（CR1），18例处于完全缓解期2（CR2），13例表现为晚期疾病（≥完全缓解期3）。患者接受了来自 HLA 相同的同胞（n = 45）或表型 HLA 相同的家族供体（n = 5）的异基因SCT。所有无病患者的中位随访时间为61个月（范围8.8 - 177个月）。在分析时，有12例患者死亡。其中11例死于复发，1例死于移植相关死亡率（TRM）。所有患者、急性髓系白血病（AML）患者（n = 12）和淋巴系统恶性肿瘤患者（n = 38）的无病生存率（DFS）分别为61.4%和82.1%。CR1、CR2和≥CR3病例的DFS率分别为89.2%、88.1%和23.1%（p < 0.05）。对于患有血液系统恶性肿瘤的儿科患者，MEL/TBI与较低的复发率相关且毒性未增加，从而带来更好的生存率。
