title: 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.
authors.affiliation: Department of Pediatrics, Chungbuk National University College of Medicine, Cheongju, South Korea.
authors.forename: Meerim
authors.initials: M
authors.lastname: Park
authors.forename: Young Ho
authors.initials: YH
authors.lastname: Lee
authors.forename: Hae-Ryong
authors.initials: HR
authors.lastname: Kang
authors.forename: Ji Won
authors.initials: JW
authors.lastname: Lee
authors.forename: Hyoung Jin
authors.initials: HJ
authors.lastname: Kang
authors.forename: Kyung Duk
authors.initials: KD
authors.lastname: Park
authors.forename: Hee Young
authors.initials: HY
authors.lastname: Shin
authors.forename: Hyo Seop
authors.initials: HS
authors.lastname: Ahn
authors.forename: Hee Jo
authors.initials: HJ
authors.lastname: Baek
authors.forename: Hoon
authors.initials: H
authors.lastname: Kook
authors.forename: Tai Ju
authors.initials: TJ
authors.lastname: Hwang
authors.forename: Jae Wook
authors.initials: JW
authors.lastname: Lee
authors.forename: Nack-Gyun
authors.initials: NG
authors.lastname: Chung
authors.forename: Bin
authors.initials: B
authors.lastname: Cho
authors.forename: Hack-Ki
authors.initials: HK
authors.lastname: Kim
authors.forename: Soo Hyun
authors.initials: SH
authors.lastname: Lee
authors.forename: Keon Hee
authors.initials: KH
authors.lastname: Yoo
authors.forename: Ki Woong
authors.initials: KW
authors.lastname: Sung
authors.forename: Hong Hoe
authors.initials: HH
authors.lastname: Koo
authors.forename: Kyung Nam
authors.initials: KN
authors.lastname: Koh
authors.forename: Ho Joon
authors.initials: HJ
authors.lastname: Im
authors.forename: Jong Jin
authors.initials: JJ
authors.lastname: Seo
authors.forename: Jun Eun
authors.initials: JE
authors.lastname: Park
authors.forename: Yeon Jung
authors.initials: YJ
authors.lastname: Lim
authors.forename: Chuhl Joo
authors.initials: CJ
authors.lastname: Lyu
authors.forename: Jae Min
authors.initials: JM
authors.lastname: Lee
authors.forename: Jeong Ok
authors.initials: JO
authors.lastname: Hah
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000628:therapy; D018952:Antigens, CD34 / Q000378:metabolism; D001855:Bone Marrow Diseases; D000080983:Bone Marrow Failure Disorders; D020739:Brain Diseases, Metabolic, Inborn / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D006680:HLA Antigens / Q000378:metabolism; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy; D007223:Infant; D008297:Male; D015999:Multivariate Analysis; D012042:Registries; D056910:Republic of Korea; D012189:Retrospective Studies; D016896:Treatment Outcome; D061349:Unrelated Donors
chemical_list: D018952:Antigens, CD34; D006680:HLA Antigens
keywords: adolescents; children; non-malignant disease; unrelated cord blood transplantation