The effect of recombinant human iduronate-2-sulfatase (Idursulfase) on growth in young patients with mucopolysaccharidosis type II.
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.
Department of Pediatrics, S. Louis Regional Children's Hospital, Cracow, Poland.
Zbigniew
Z
Żuber
Anthropology Laboratory, The Children's Memorial Health Institute, Warsaw, Poland.
Agnieszka
A
Różdżyńska-Świątkowska
Department of Molecular Biology, University of Gdańsk, Gdańsk, Poland ; Department of Medical Genetics, The Children's Memorial Health Institute, Warsaw, Poland.
Agnieszka
A
Jurecka
Department of Pediatrics, Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
Anna
A
Tylki-Szymańska
D002648:Child; D002675:Child, Preschool; D003710:Demography; D056947:Enzyme Replacement Therapy; D048788:Growth and Development / Q000187:drug effects*; D006801:Humans; D007066:Iduronate Sulfatase / Q000494:pharmacology* / Q000627:therapeutic use*; D016532:Mucopolysaccharidosis II / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000494:pharmacology* / Q000627:therapeutic use*
D011994:Recombinant Proteins; D007066:Iduronate Sulfatase; C517982:idursulfase