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The effect of recombinant human iduronate-2-sulfatase (Idursulfase) on growth in young patients with mucopolysaccharidosis type II.

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.
AuthorsZbigniew Żuber, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Anna Tylki-Szymańska
JournalPloS one (PLoS One) Vol. 9 Issue 1 Pg. e85074 ( 2014) ISSN: 1932-6203 [Electronic] United States
PMID24454794 (Publication Type: Journal Article)
Chemical References
  • Recombinant Proteins
  • Iduronate Sulfatase
  • idursulfase
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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