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.
Department of Metabolic Diseases, Endocrinology and Diabetology, The Children's Memorial Health Institute, Al. Dzieci Polskich 20, 04730 Warsaw, Poland.
Anna
A
Tylki-Szymanska
Agnieszka
A
Rozdzynska
Agnieszka
A
Jurecka
Jolanta
J
Marucha
Barbara
B
Czartoryska
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