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

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...

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Autores principales: Cho, Sung Yoon, Huh, Rimm, Chang, Mi Sun, Lee, Jieun, Kwun, Younghee, Maeng, Se Hyun, Kim, Su Jin, Sohn, Young Bae, Park, Sung Won, Kwon, Eun-Kyung, Han, Sun Ju, Jung, Jooyoun, Jin, Dong-Kyu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Academy of Medical Sciences 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3924006/
https://www.ncbi.nlm.nih.gov/pubmed/24550654
http://dx.doi.org/10.3346/jkms.2014.29.2.254
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author Cho, Sung Yoon
Huh, Rimm
Chang, Mi Sun
Lee, Jieun
Kwun, Younghee
Maeng, Se Hyun
Kim, Su Jin
Sohn, Young Bae
Park, Sung Won
Kwon, Eun-Kyung
Han, Sun Ju
Jung, Jooyoun
Jin, Dong-Kyu
author_facet Cho, Sung Yoon
Huh, Rimm
Chang, Mi Sun
Lee, Jieun
Kwun, Younghee
Maeng, Se Hyun
Kim, Su Jin
Sohn, Young Bae
Park, Sung Won
Kwon, Eun-Kyung
Han, Sun Ju
Jung, Jooyoun
Jin, Dong-Kyu
author_sort Cho, Sung Yoon
collection PubMed
description 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. GRAPHICAL ABSTRACT: [Image: see text]
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spelling pubmed-39240062014-02-18 Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome) Cho, Sung Yoon Huh, Rimm Chang, Mi Sun Lee, Jieun Kwun, Younghee Maeng, Se Hyun Kim, Su Jin Sohn, Young Bae Park, Sung Won Kwon, Eun-Kyung Han, Sun Ju Jung, Jooyoun Jin, Dong-Kyu J Korean Med Sci Original Article 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. GRAPHICAL ABSTRACT: [Image: see text] The Korean Academy of Medical Sciences 2014-02 2014-01-28 /pmc/articles/PMC3924006/ /pubmed/24550654 http://dx.doi.org/10.3346/jkms.2014.29.2.254 Text en © 2014 The Korean Academy of Medical Sciences. http://creativecommons.org/licenses/by-nc/3.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Article
Cho, Sung Yoon
Huh, Rimm
Chang, Mi Sun
Lee, Jieun
Kwun, Younghee
Maeng, Se Hyun
Kim, Su Jin
Sohn, Young Bae
Park, Sung Won
Kwon, Eun-Kyung
Han, Sun Ju
Jung, Jooyoun
Jin, Dong-Kyu
Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title_full Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title_fullStr Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title_full_unstemmed Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title_short Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
title_sort impact of enzyme replacement therapy on linear growth in korean patients with mucopolysaccharidosis type ii (hunter syndrome)
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3924006/
https://www.ncbi.nlm.nih.gov/pubmed/24550654
http://dx.doi.org/10.3346/jkms.2014.29.2.254
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