Unrelated donor cord blood transplantation for non-malignant disorders in children and adolescents.

Abstract:

:This study analyzes the data reported to the Korean Cord Blood Registry between 1994 and 2008, involving children and adolescents with non-malignant diseases. Sixty-five patients were evaluated in this study: SAA (n = 24), iBMFS, (n = 16), and primary immune deficiency/inherited metabolic disorder (n = 25). The CI of neutrophil recovery was 73.3% on day 42. By day 100, the CI of acute grade II-IV graft-versus-host disease was 32.3%. At a median follow-up of 71 months, five-yr OS was 50.7%. The survival rate (37.5%) and CI of neutrophil engraftment (37.5%) were lowest in patients with iBMFS. Deaths were mainly due to infection, pulmonary complications, and hemorrhage. In a multivariate analysis, the presence of >3.91 × 10(5) /kg of infused CD34 + cells was the only factor consistently identified as significantly associated with neutrophil engraftment (p = 0.04) and OS (p = 0.03). UCBT using optimal cell doses appears to be a feasible therapy for non-malignant diseases in children and adolescents for whom there is no appropriate HLA-matched related donor. Strategies to reduce transplant-related toxicities would improve the outcomes of UCBT in non-malignant diseases.

journal_name

Pediatr Transplant

authors

Park M,Lee YH,Kang HR,Lee JW,Kang HJ,Park KD,Shin HY,Ahn HS,Baek HJ,Kook H,Hwang TJ,Lee JW,Chung NG,Cho B,Kim HK,Lee SH,Yoo KH,Sung KW,Koo HH,Koh KN,Im HJ,Seo JJ,Park JE,Lim YJ,Lyu CJ,Lee JM,Hah JO,K

doi

10.1111/petr.12213

subject

Has Abstract

pub_date

2014-03-01 00:00:00

pages

221-9

issue

2

eissn

1397-3142

issn

1399-3046

journal_volume

18

pub_type

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