Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome).
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| 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.
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| Authors | Sung Yoon Cho, 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 |
| Journal | Journal of Korean medical science (J Korean Med Sci) Vol. 29 Issue 2 Pg. 254-60 (Feb 2014) ISSN: 1598-6357 [Electronic] Korea (South) |
| PMID | 24550654 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't) |
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