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


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.
Tylki-Szymanska
Anna
A
Department of Metabolic Diseases, Endocrinology and Diabetology, The Children's Memorial Health Institute, Al. Dzieci Polskich 20, 04730 Warsaw, Poland.
Rozdzynska
Agnieszka
A
Jurecka
Agnieszka
A
Marucha
Jolanta
J
Czartoryska
Barbara
B
D000293:Adolescent; D000886:Anthropometry; D001827:Body Height / Q000187:drug effects*; D001835:Body Weight / Q000187:drug effects*; D002648:Child; D002675:Child, Preschool; D056947:Enzyme Replacement Therapy; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007068:Iduronidase / Q000235:genetics / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D008059:Mucopolysaccharidosis I / Q000188:drug therapy* / Q000473:pathology; D011994:Recombinant Proteins / Q000627:therapeutic use; D012189:Retrospective Studies; D016896:Treatment Outcome
D011994:Recombinant Proteins; D007068:Iduronidase