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KMID : 0191120140290020254
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Journal of Korean Medical Science
2014 Volume.29 No. 2 p.254 ~ p.260
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Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
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Cho Sung-Yoon
Huh Rimm
Chang Mi-Sun
Lee Ji-Eun
Kwun Young-Hee
Maeng Se-Hyun
Kim Su-Jin
Sohn Young-Bae
Park Sung-Won
Kwon Eun-Kyung
Han Sun-Ju
Jung Joo-youn
Jin Dong-Kyu
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Abstract
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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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KEYWORD
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Mucopolysaccharidosis II, Hunter Syndrome, Enzyme Replacement Therapy, Growth, Elaprase, Hunterase
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