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Unrelated donor cord blood transplantation for non‐malignant disorders in children and adolescents
Author(s) -
Park Meerim,
Lee Young Ho,
Kang HaeRyong,
Lee Ji Won,
Kang Hyoung Jin,
Park Kyung Duk,
Shin Hee Young,
Ahn Hyo Seop,
Baek Hee Jo,
Kook Hoon,
Hwang Tai Ju,
Lee Jae Wook,
Chung NackGyun,
Cho Bin,
Kim HackKi,
Lee Soo Hyun,
Yoo Keon Hee,
Sung Ki Woong,
Koo Hong Hoe,
Koh Kyung Nam,
Im Ho Joon,
Seo Jong Jin,
Park Jun Eun,
Lim Yeon Jung,
Lyu Chuhl Joo,
Lee Jae Min,
Hah Jeong Ok
Publication year - 2014
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12213
Subject(s) - medicine , cord blood , multivariate analysis , transplantation , gastroenterology , pediatrics , immunology
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.