Locatelli F, Rocha V, Chastang C, Arcese W, Michel G, Abecasis M, Messina C, Ortega J, Badell-Serra I, Plouvier E, Souillet G, Jouet J P, Pasquini R, Ferreira E, Garnier F, Gluckman E
Clinica Pediatrica, IRCCS Policlinico San Matteo, University of Pavia, Italy.
Blood. 1999 Jun 1;93(11):3662-71.
We have analyzed factors influencing the outcome of 102 children with acute leukemia given a cord blood transplantation (CBT) and reported to the Eurocord Registry. Seventy patients with acute lymphoblastic and 32 with acute myeloid leukemia were given either a related (n = 42) or an unrelated (n = 60) CBT. Children given CBT during first or second complete remission were considered as belonging to the good-risk group (n = 66), whereas those who received a transplant in a more advanced stage of disease were assigned to the poor-risk group (n = 36). In the related group (RCBT), 12 of 42 patients received transplantation from an HLA-disparate donor, whereas in the unrelated group (UCBT) 54 of 60 received an HLA mismatched CBT. Kaplan-Meier estimates for neutrophil recovery at day 60 were 84% +/- 7% in RCBT and 79 +/- 6% in UCBT (P =.16). In multivariate analysis, the most important factor influencing neutrophil engraftment in UCBT was a nucleated cell dose infused greater than 3.7 x 10(7)/kg (P =.05, relative risk [RR] of 1.85, 95% confidence interval [CI]: 0.98-3.4). The incidence of grade II through IV acute graft-versus-host disease was 41% +/- 8% in the RCBT group and 37% +/- 6% in the UCBT group (P =.59). Kaplan-Meier estimates of 2-year event-free survival (EFS) after RCBT or UCBT were 39% +/- 8% and 30% +/- 7%, respectively (P =.19). In multivariate analysis, the most important factor influencing EFS was disease status at time of transplantation: good-risk patients had a 2-year EFS of 49% +/- 7% as compared to 8% +/- 5% in patients with more advanced disease (P =.0003, RR: 0.40, 95% CI: 0.24 to 0. 65). This was a consequence of both an increased 1-year transplant related mortality and a higher 2-year relapse rate in the poor-risk group (65% +/- 9% and 77% +/- 14%, respectively), as compared with good risk patients (34% +/- 6% and 31% +/- 9%, respectively). These data confirm that allogeneic CBT from either a related or an unrelated donor is a feasible procedure able to cure a significant proportion of children with acute leukemia, especially if transplanted in a favorable phase of disease.
我们分析了影响102例接受脐血移植（CBT）的急性白血病患儿预后的因素，并向欧洲脐血库登记处报告。70例急性淋巴细胞白血病患儿和32例急性髓细胞白血病患儿接受了相关供者（n = 42）或无关供者（n = 60）的CBT。在首次或第二次完全缓解期接受CBT的患儿被视为低危组（n = 66），而在疾病更晚期接受移植的患儿被归为高危组（n = 36）。在相关供者组（RCBT）中，42例患者中有12例接受了HLA配型不合供者的移植，而在无关供者组（UCBT）中，60例患者中有54例接受了HLA配型不相合的CBT。RCBT组60天时中性粒细胞恢复的Kaplan-Meier估计值为84%±7%，UCBT组为79%±6%（P = 0.16）。多因素分析显示，影响UCBT中性粒细胞植入的最重要因素是输入的有核细胞剂量大于3.7×10⁷/kg（P = 0.05，相对危险度[RR]为1.85，95%置信区间[CI]：0.98 - 3.4）。RCBT组Ⅱ至Ⅳ级急性移植物抗宿主病的发生率为41%±8%，UCBT组为37%±6%（P = 0.59）。RCBT或UCBT后2年无事件生存率（EFS）的Kaplan-Meier估计值分别为39%±8%和30%±7%（P = 0.19）。多因素分析显示，影响EFS的最重要因素是移植时的疾病状态：低危患者2年EFS为49%±7%，而疾病更晚期患者为8%±5%（P = 0.0003，RR：0.40，95%CI：0.24至0.65）。这是由于高危组1年移植相关死亡率增加和2年复发率更高（分别为65%±9%和77%±14%），而低危患者分别为34%±6%和31%±9%。这些数据证实，来自相关或无关供者的异基因CBT是一种可行的治疗方法，能够治愈相当比例的急性白血病患儿，尤其是在疾病的有利阶段进行移植时。
