Ballen Karen, Logan Brent R, Chitphakdithai Pintip, Kuxhausen Michelle, Spellman Stephen R, Adams Alexia, Drexler Rebecca J, Duffy Merry, Kemp Ann, King Roberta, Babic Aleksandar, Delaney Colleen, Karanes Chatchada, Kurtzberg Joanne, Petz Lawrence, Scaradavou Andromachi, Shpall Elizabeth J, Smith Clayton, Confer Dennis L, Miller John P
University of Virginia Health Center, Charlottesville, Virginia.
Center for International Blood and Marrow Transplant Research, Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin.
Biol Blood Marrow Transplant. 2020 Apr;26(4):745-757. doi: 10.1016/j.bbmt.2019.11.016. Epub 2019 Nov 19.
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/mm) 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.
脐带血（UCB）移植（UCBT）是治疗血液系统恶性肿瘤和遗传性疾病患者的一种有效方法，扩大了无法找到完全匹配无关供体的非白种人患者接受移植的机会。2011年，美国食品药品监督管理局要求无关供体的UCBT使用已获许可的UCB或根据研究性新药（IND）计划使用未获许可的UCB进行。国家骨髓捐献计划管理一项IND，在该计划下，2456例患者（1499例成人和957例儿童，564例患有恶性疾病，393例患有非恶性疾病）在2011年10月至2016年12月期间接受了单倍体或双倍体UCBT。患者中位年龄为31岁（范围为<1至81岁），50%的儿童和36%的成人是非白种人。成人中性粒细胞植入的中位时间（即绝对中性粒细胞计数≥500/mm）为22天，患有恶性疾病的儿科患者为20天，患有非恶性疾病的儿科患者为19天，42天时相应的植入率分别为89%、88%和90%。在这三组患者中，急性移植物抗宿主病（GVHD）II-IV级的发生率分别为35%、32%和24%；慢性GVHD的发生率分别为24%、26%和24%；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、化疗敏感的早期疾病以及较高的体能状态评分可预测成人OS改善。在接受单倍体UCBT的恶性肿瘤儿童亚组分析中，使用已获许可的UCB（n = 48）或未获许可的UCB（n = 382）与相似的植入和生存率相关。使用未获许可的UCB单位是安全有效的，为不同人群提供了重要的移植物来源。
