Abstract |
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked, recessive, lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase. Early bone involvement leads to decreased growth velocity and short stature in nearly all patients. Our analysis aimed to investigate the effects of enzyme replacement therapy (ERT) with idursulfase ( Elaprase) on growth in young patients with mucopolysaccharidosis type II. Analysis of longitudinal anthropometric data of MPS II patients (group 1, n = 13) who started ERT before 6 years of age (range from 3 months to 6 years, mean 3.6 years, median 4 years) was performed and then compared with retrospective analysis of data for MPS II patients naïve to ERT (group 2, n = 50). Patients in group 1 received intravenous idursulfase at a standard dose of 0.58 mg/kg weekly for 52-288 weeks. The course of average growth curve for group 1 was very similar to growth pattern in group 2. The average value of body height in subsequent years in group 1 was a little greater than in group 2, however, the difference was not statistically significant. In studied patients with MPS II, idursulfase did not appear to alter the growth patterns.
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Authors | Zbigniew Żuber, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Anna Tylki-Szymańska |
Journal | PloS one
(PLoS One)
Vol. 9
Issue 1
Pg. e85074
( 2014)
ISSN: 1932-6203 [Electronic] United States |
PMID | 24454794
(Publication Type: Journal Article)
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Chemical References |
- Recombinant Proteins
- Iduronate Sulfatase
- idursulfase
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Topics |
- Child
- Child, Preschool
- Demography
- Enzyme Replacement Therapy
- Growth and Development
(drug effects)
- Humans
- Iduronate Sulfatase
(pharmacology, therapeutic use)
- Mucopolysaccharidosis II
(drug therapy)
- Recombinant Proteins
(pharmacology, therapeutic use)
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