Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome).
10.3346/jkms.2014.29.2.254
- Author:
Sung Yoon CHO
1
;
Rimm HUH
;
Mi Sun CHANG
;
Jieun LEE
;
Younghee KWUN
;
Se Hyun MAENG
;
Su Jin KIM
;
Young Bae SOHN
;
Sung Won PARK
;
Eun Kyung KWON
;
Sun Ju HAN
;
Jooyoun JUNG
;
Dong Kyu JIN
Author Information
1. Department of Pediatrics, Hanyang University Guri Hopistal, Hanyang University College of Medicine, Guri, Korea.
- Publication Type:Original Article ; Research Support, Non-U.S. Gov't
- Keywords:
Mucopolysaccharidosis II;
Hunter Syndrome;
Enzyme Replacement Therapy;
Growth;
Elaprase;
Hunterase
- MeSH:
Adolescent;
Body Height;
Child;
Child, Preschool;
Demography;
Enzyme Replacement Therapy;
Humans;
Iduronate Sulfatase/*therapeutic use;
Infant;
Male;
Mild Cognitive Impairment/etiology;
Mucopolysaccharidosis II/complications/diagnosis/*therapy;
Mutation;
Phenotype;
Protein Isoforms/therapeutic use;
Republic of Korea;
Young Adult
- From:Journal of Korean Medical Science
2014;29(2):254-260
- CountryRepublic of Korea
- Language:English
-
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(R)) or idursulfase beta (Hunterase(R)) 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.
