NDLI: Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)

Content Provider	PubMed Central
Author	Cho, Sung Yoon Huh, Rimm Chang, Mi Sun Lee, Jieun Kwun, Younghee Hyun, Maeng Se Kim, Su Jin Sohn, Young Bae Park, Sung Won Kwon, Eun-kyung Han, Sun Ju Jung, Jooyoun Jin, Dong-kyu
Copyright Year	2014
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.
Related Links	http://dx.doi.org/10.3346/jkms.2014.29.2.254
Starting Page	254
File Format	PDF
ISSN	10118934
e-ISSN	15986357
Journal	Journal of Korean Medical Science
Issue Number	2
Volume Number	29
Language	English
Publisher	The Korean Academy of Medical Sciences
Publisher Date	2014-01-01
Access Restriction	Open
Rights Holder	The Korean Academy of Medical Sciences
Subject Keyword	Medicine(all) Research in Higher Education
Content Type	Text
Resource Type	Article
Subject	Medicine

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