Abstract | OBJECTIVES: 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 52-260 weeks 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 100 U/kg (0.58 mg/kg) weekly for 52-260 weeks. 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 96-260 weeks 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.
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Authors | Anna Tylki-Szymanska, Agnieszka Rozdzynska, Agnieszka Jurecka, Jolanta Marucha, Barbara Czartoryska |
Journal | Molecular genetics and metabolism
(Mol Genet Metab)
Vol. 99
Issue 1
Pg. 10-7
(Jan 2010)
ISSN: 1096-7206 [Electronic] United States |
PMID | 19783188
(Publication Type: Clinical Trial, Journal Article)
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Chemical References |
- Recombinant Proteins
- Iduronidase
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Topics |
- Adolescent
- Anthropometry
- Body Height
(drug effects)
- Body Weight
(drug effects)
- Child
- Child, Preschool
- Enzyme Replacement Therapy
- Female
- Follow-Up Studies
- Humans
- Iduronidase
(genetics, therapeutic use)
- Infant
- Male
- Mucopolysaccharidosis I
(drug therapy, pathology)
- Recombinant Proteins
(therapeutic use)
- Retrospective Studies
- Treatment Outcome
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