title: Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome).
abstract: Hunter syndrome (or mucopolysaccharidosis type II [MPS II]) arises because of a deficiency in the lysosomal enzyme iduronate-2-sulfatase. Short stature is a prominent and consistent feature in MPS II. Enzyme replacement therapy (ERT) with idursulfase (Elaprase®) or idursulfase beta (Hunterase®) have been developed for these patients. The effect of ERT on the growth of Korean patients with Hunter syndrome was evaluated at a single center. This study comprised 32 patients, who had received ERT for at least 2 yr; they were divided into three groups according to their ages at the start of ERT: group 1 (<6 yr, n=14), group 2 (6-10 yr, n=11), and group 3 (10-20 yr, n=7). The patients showed marked growth retardation as they got older. ERT may have less effect on the growth of patients with the severe form of Hunter syndrome. The height z-scores in groups 2 and 3 revealed a significant change (the estimated slopes before and after the treatment were -0.047 and -0.007, respectively: difference in the slope, 0.04; P<0.001). Growth in response to ERT could be an important treatment outcome or an endpoint for future studies.
authors.affiliation: Department of Pediatrics, Hanyang University Guri Hopistal, Hanyang University College of Medicine, Guri, Korea.
authors.forename: Sung Yoon
authors.initials: SY
authors.lastname: Cho
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Rimm
authors.initials: R
authors.lastname: Huh
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Mi Sun
authors.initials: MS
authors.lastname: Chang
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Jieun
authors.initials: J
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Younghee
authors.initials: Y
authors.lastname: Kwun
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Se Hyun
authors.initials: SH
authors.lastname: Maeng
authors.affiliation: Department of Pediatrics, Kwandong University College of Medicine, Myongji Hospital, Goyang, Korea.
authors.forename: Su Jin
authors.initials: SJ
authors.lastname: Kim
authors.affiliation: Department of Medical Genetics, Ajou University Hospital, Ajou University School of Medicine, Suwon, Korea.
authors.forename: Young Bae
authors.initials: YB
authors.lastname: Sohn
authors.affiliation: Department of Pediatrics, Kwandong University College of Medicine, Cheil General Hospital & Woman's Health care Center, Seoul, Korea.
authors.forename: Sung Won
authors.initials: SW
authors.lastname: Park
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Eun-Kyung
authors.initials: EK
authors.lastname: Kwon
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Sun Ju
authors.initials: SJ
authors.lastname: Han
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Jooyoun
authors.initials: J
authors.lastname: Jung
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Dong-Kyu
authors.initials: DK
authors.lastname: Jin
mesh_terms: D000293:Adolescent; D001827:Body Height; D002648:Child; D002675:Child, Preschool; D060825:Cognitive Dysfunction / Q000209:etiology; D003710:Demography; D056947:Enzyme Replacement Therapy; D006801:Humans; D007066:Iduronate Sulfatase / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D016532:Mucopolysaccharidosis II / Q000150:complications / Q000175:diagnosis / Q000628:therapy*; D009154:Mutation; D010641:Phenotype; D020033:Protein Isoforms / Q000627:therapeutic use; D056910:Republic of Korea; D055815:Young Adult
chemical_list: D020033:Protein Isoforms; D007066:Iduronate Sulfatase; C517982:idursulfase
keywords: Elaprase; Enzyme Replacement Therapy; Growth; Hunter Syndrome; Hunterase; Mucopolysaccharidosis II