Volume 18, Issue 2 pp. 221-229
Original Article

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

Meerim Park

Meerim Park

Department of Pediatrics, Chungbuk National University College of Medicine, Cheongju, South Korea

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Young Ho Lee

Corresponding Author

Young Ho Lee

Department of Pediatrics, Hanyang University Medical Center, Hanyang University College of Medicine, Seoul, South Korea

Young Ho Lee, Department of Pediatrics, Hanyang University Medical Center, Hanyang University College of Medicine, Wangsimni-ro, 222, Seongdong-gu, Seoul, Korea

Tel.: 82 2 2290 8383

Fax: 82 2 2297 2380

E-mail: [email protected]

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Hae-Ryong Kang

Hae-Ryong Kang

Department of Pediatrics, Hanyang University Medical Center, Hanyang University College of Medicine, Seoul, South Korea

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Ji Won Lee

Ji Won Lee

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, South Korea

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Hyoung Jin Kang

Hyoung Jin Kang

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, South Korea

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Kyung Duk Park

Kyung Duk Park

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, South Korea

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Hee Young Shin

Hee Young Shin

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, South Korea

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Hyo Seop Ahn

Hyo Seop Ahn

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, South Korea

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Hee Jo Baek

Hee Jo Baek

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Gwangju, South Korea

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Hoon Kook

Hoon Kook

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Gwangju, South Korea

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Tai Ju Hwang

Tai Ju Hwang

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Gwangju, South Korea

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Jae Wook Lee

Jae Wook Lee

Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, South Korea

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Nack-Gyun Chung

Nack-Gyun Chung

Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, South Korea

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Bin Cho

Bin Cho

Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, South Korea

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Hack-Ki Kim

Hack-Ki Kim

Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, South Korea

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Soo Hyun Lee

Soo Hyun Lee

Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

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Keon Hee Yoo

Keon Hee Yoo

Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

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Ki Woong Sung

Ki Woong Sung

Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

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Hong Hoe Koo

Hong Hoe Koo

Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

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Kyung Nam Koh

Kyung Nam Koh

Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, South Korea

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Ho Joon Im

Ho Joon Im

Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, South Korea

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Jong Jin Seo

Jong Jin Seo

Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, South Korea

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Jun Eun Park

Jun Eun Park

Department of Pediatrics, Ajou University School of Medicine, Suwon, South Korea

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Yeon Jung Lim

Yeon Jung Lim

Department of Pediatrics, Chungnam National University College of Medicine, Daejon, South Korea

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Chuhl Joo Lyu

Chuhl Joo Lyu

Department of Pediatrics, Yonsei University, College of Medicine, Seoul, South Korea

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Jae Min Lee

Jae Min Lee

Department of Pediatrics, Yeungnam University College of Medicine, Daegu, South Korea

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Jeong Ok Hah

Jeong Ok Hah

Department of Pediatrics, Yeungnam University College of Medicine, Daegu, South Korea

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on behalf of the Korean Cord Blood Transplantation Working Party

on behalf of the Korean Cord Blood Transplantation Working Party

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First published: 30 December 2013
Citations: 10

[Correction added on 3rd January 2014, after first online publication: Jae Min Lee has been added to the author's list.]

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 × 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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