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Anthropometric data of 14 patients with mucopolysaccharidosis I: retrospective analysis and efficacy of recombinant human alpha-L-iduronidase (laronidase).

AbstractOBJECTIVES:
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 52-260 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.
AuthorsAnna Tylki-Szymanska, Agnieszka Rozdzynska, Agnieszka Jurecka, Jolanta Marucha, Barbara Czartoryska
JournalMolecular genetics and metabolism (Mol Genet Metab) Vol. 99 Issue 1 Pg. 10-7 (Jan 2010) ISSN: 1096-7206 [Electronic] United States
PMID19783188 (Publication Type: Clinical Trial, Journal Article)
Chemical References
  • Recombinant Proteins
  • Iduronidase
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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