Umbilical cord blood (UCB) transplantation (UCBT) is a curative procedure for patients with hematologic malignancies and genetic disorders and expands access to transplantation for non-Caucasian patients unable to find a fully matched unrelated donor. In 2011, the US Food and Drug Administration required that unrelated UCBT be performed using either licensed UCB or unlicensed UCB under the Investigational New Drug (IND) program. The National Marrow Donor Program manages an IND under which 2456 patients (1499 adults and 957 children, 564 with malignant diseases and 393 with nonmalignant diseases) underwent single or double UCBT between October 2011 and December 2016. The median patient age was 31 years (range, <1 to 81 years), and 50% of children and 36% of adults were non-Caucasian. The median time to neutrophil engraftment (ie, absolute neutrophil count ≥500/mm3) was 22 days for adults, 20 days for pediatric patients with malignant diseases, and 19 days for pediatric patients with nonmalignant diseases, with corresponding rates of engraftment at 42 days of 89%, 88%, and 90%. In these 3 groups of patients, the incidence of acute graft-versus-host disease (GVHD) grade II-IV was 35%, 32%, and 24%; the incidence of chronic GVHD was 24%, 26%, and 24%; and 1-year overall survival (OS) was 57%, 71%, and 79%, respectively. In multivariate analysis, younger age, lower Hematopoietic Cell Transplantation-Specific Comorbidity Index, early-stage chemotherapy-sensitive disease, and higher performance score were predictive of improved OS for adults. In a subset analysis of children with malignancies undergoing single UCBT, the use of either licensed UCB (n = 48) or unlicensed UCB (n = 382) was associated with similar engraftment and survival. The use of unlicensed UCB units is safe and effective and provides an important graft source for a diverse population.

中文翻译：

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未经许可的脐带血单位为不同人群提供了安全有效的移植物来源：对2456名脐带血接受者的研究。

脐带血（UCB）移植（UCBT）是血液系统恶性肿瘤和遗传疾病患者的治愈方法，并为无法找到完全匹配的无关供体的非高加索患者提供了获得移植的机会。在2011年，美国食品药品监督管理局要求根据研究新药（IND）计划，使用获得许可的UCB或未经许可的UCB进行无关的UCBT。国家骨髓捐献者计划管理着一项IND，根据该IND，2011年10月至2016年12月，单次或双次UCBT接受了2456例患者（1499名成人和957名儿童，564例恶性疾病和393例非恶性疾病）。平均患者年龄为31岁（范围，<1至81岁），并且50％的儿童和36％的成年人是非高加索人。中性粒细胞植入的中位时间（即 中性粒细胞绝对计数≥500/ mm3）成人为22天，小儿恶性疾病患者为20天，小儿非恶性疾病患者为19天，在42天的相应植入率分别为89％，88％和90 ％。在这3组患者中，II-IV级急性移植物抗宿主病（GVHD）的发生率分别为35％，32％和24％。慢性GVHD的发生率分别为24％，26％和24％；和1年总生存率（OS）分别为57％，71％和79％。在多变量分析中，年龄较小，造血细胞移植特异性合并症指数较低，早期化疗敏感性疾病和较高的性能评分可预示成人OS改善。在接受单次UCBT的恶性肿瘤儿童的子集分析中，使用有执照的UCB（n = 48）或无执照的UCB（n = 382）与相似的植入和存活率相关。使用未经许可的UCB装置是安全有效的，并为不同人群提供了重要的移植物来源。