Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients. Biol Blood Marrow Transplant 2020 Apr;26(4):745-757
Date
11/23/2019Pubmed ID
31756539Pubmed Central ID
PMC7198366DOI
10.1016/j.bbmt.2019.11.016Scopus ID
2-s2.0-85077166820 (requires institutional sign-in at Scopus site) 11 CitationsAbstract
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.
Author List
Ballen K, Logan BR, Chitphakdithai P, Kuxhausen M, Spellman SR, Adams A, Drexler RJ, Duffy M, Kemp A, King R, Babic A, Delaney C, Karanes C, Kurtzberg J, Petz L, Scaradavou A, Shpall EJ, Smith C, Confer DL, Miller JPAuthor
Brent R. Logan PhD Director, Professor in the Institute for Health and Equity department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdolescentAdult
Aged
Aged, 80 and over
Child
Child, Preschool
Cord Blood Stem Cell Transplantation
Fetal Blood
Graft vs Host Disease
Hematologic Neoplasms
Hematopoietic Stem Cell Transplantation
Humans
Infant
Middle Aged
Young Adult