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Umbilical Cord Blood Transplantation: Challenges and Future Directions.

ABSTRACT: Since the first successful allogeneic transplants performed in Seattle 50 years ago, the field of transplantation has evolved considerably, with improvements in human leukocyte antigen typing, patient selection, reduced intensity regimens, and graft-versus-host disease prophylaxis. A major breakthrough has been the availability of more donor options, first via the National Marrow Donor Program-Be the Match [Biol Blood Marrow Transplant 2008;14:2-7]. Then, in the 1990s, unrelated umbilical cord blood transplantation became available, first for children and then for adults [New Engl J Med 1996;35:157-166]. More recently mismatched unrelated transplants and haploidentical donor options became available [Blood 2011;118:282-288]. In 2017, there is a donor for almost every patient who needs a transplant. In this review, we will discuss the state of the science (and art) of cord blood transplant, focusing on successes, challenges, and future directions. Stem Cells Translational Medicine 2017;6:1312-1315.

SUBMITTER: Ballen K

PROVIDER: S-EPMC5442719 | biostudies-literature | 2017 May

REPOSITORIES: biostudies-literature

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Umbilical Cord Blood Transplantation: Challenges and Future Directions.

Ballen Karen K

Stem cells translational medicine 20170501 5

Since the first successful allogeneic transplants performed in Seattle 50 years ago, the field of transplantation has evolved considerably, with improvements in human leukocyte antigen typing, patient selection, reduced intensity regimens, and graft-versus-host disease prophylaxis. A major breakthrough has been the availability of more donor options, first via the National Marrow Donor Program-Be the Match [Biol Blood Marrow Transplant 2008;14:2-7]. Then, in the 1990s, unrelated umbilical cord b ...[more]

PMID: 28456009

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Project description:For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.

Dataset Information

Umbilical Cord Blood Transplantation: Challenges and Future Directions.

Publications

Umbilical Cord Blood Transplantation: Challenges and Future Directions.

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