| Title | Anthropometric data of 14 patients with mucopolysaccharidosis I: Retrospective analysis and efficacy of recombinant human alpha-l-iduronidase (laronidase). | | Author(s) | Tylki-Szymanska A, Rozdzynska A, Jurecka A, Marucha J, Czartoryska B | | Institution | Department of Metabolic Diseases, Endocrinology and Diabetology, The Children's Memorial Health Institute, Al. Dzieci Polskich 20, 04730 Warsaw, Poland. | | Source | Mol Genet Metab 2009 Aug 27. | | Abstract | Objectives: Our goal was to evaluate growth patterns in terms of body height, weight, head and chest circumference in patients with mucopolysaccharidosis type I (MPS I) without treatment and after enzyme replacement therapy (ERT) with alpha-l-iduronidase (laronidase). Patients and methods: Anthropometric features of 14 patients with MPS I were followed from birth until the introduction of ERT (group 1-1st year of life, group 2 3rd year of life), after 52-260weeks of ERT and periodically during treatment. The data since birth until beginning of treatment was obtained by retrospective review of patients' charts. Patients received intravenous laronidase at 100U/kg (0.58mg/kg) weekly for 52-260weeks.
Results: Patients from group 1 (n=7) and group 2 (n=7) had similar characteristics at the time of birth but showed significant difference when compared with healthy population. Growth patterns were associated significantly with the MPS I at birth. After 96-260weeks of ERT, patients receiving laronidase (group 1) compared with group 2 did not show statistically significant improvement.
Conclusions: Anthropometric features of patients with MPS I significantly differ from the healthy population. Children with MPS I grew considerably slower, and differences between healthy and affected children increased with age. In studied patients with MPS I, laronidase did not appear to alter the growth patterns. | | Language | ENG | | Pub Type(s) | JOURNAL ARTICLE
| | PubMed ID | 19783188 |
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