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

 Meerim Park  ;  Young Ho Lee  ;  Hae-Ryong Kang  ;  Ji Won Lee  ;  Hyoung Jin Kang  ;  Kyung Duk Park  ;  Hee Young Shin  ;  Hyo Seop Ahn  ;  Hee Jo Baek  ;  Hoon Kook  ;  Tai Ju Hwang  ;  Jae Wook Lee  ;  Nack-Gyun Chung  ;  Bin Cho  ;  Hack-Ki Kim  ;  Soo Hyun Lee  ;  Keon Hee Yoo  ;  Ki Woong Sung  ;  Hong Hoe Koo  ;  Kyung Nam Koh  ;  Ho Joon Im  ;  Jong Jin Seo  ;  Jun Eun Park  ;  Yeon Jung Lim  ;  Chuhl Joo Lyu  ;  Jae Min Lee  ;  Jeong Ok Hah 
 Pediatric Transplantation, Vol.18(2) : 221-229, 2014 
Journal Title
 Pediatric Transplantation 
Issue Date
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 × 105/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.
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1. College of Medicine (의과대학) > Dept. of Pediatrics (소아청소년과학교실) > 1. Journal Papers
Yonsei Authors
유철주(Lyu, Chuhl Joo) ORCID logo https://orcid.org/0000-0001-7124-7818
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