Document Detail


Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
MedLine Citation:
PMID:  20634905     Owner:  NLM     Status:  Publisher    
Abstract/OtherAbstract:
BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3 Extension clinical trials. Height, weight, and Tanner stage data were collected. Pooled data were analyzed to determine mean height increase by treatment week, growth impacts of pubertal status, baseline urinary GAG, and age at treatment initiation. Growth rate for approximately 2 years prior to and following treatment initiation was analyzed using longitudinal modeling. RESULTS: Mean height increased by 2.9 cm after 48 weeks and 4.3 cm after 96 weeks on enzyme replacement therapy (ERT). Growth on ERT was not correlated with baseline urinary GAG. Patients under 16 years of age showed greatest increases in height on treatment. Model results based on pooled data showed significant improvement in growth rate during 96 weeks of ERT when compared to the equivalent pretreatment time period. Delayed pubertal onset or progression was noted in 10 patients entering the clinical trials; all of whom showed progression of at least one Tanner stage during 2 years on ERT, and 6 of whom (60%) completed puberty. CONCLUSION: Analysis of mean height by treatment week and longitudinal modeling demonstrate significant increase in height and growth rate in MPS VI patients receiving long-term ERT. This impact was greatest in patients aged below 16 years. Height increase may result from bone growth and/or reduction in joint contractures. Bone growth and resolution of delayed puberty may be related to improvements in general health, bone cell health, nutrition, endocrine gland function and reduced inflammation.
Authors:
Celeste Decker; Zi-Fan Yu; Roberto Giugliani; Ida Vanessa D Schwartz; Nathalie Guffon; Elisa Leão Teles; M Clara Sá Miranda; J Edmond Wraith; Michael Beck; Laila Arash; Maurizio Scarpa; David Ketteridge; John J Hopwood; Barbara Plecko; Robert Steiner; Chester B Whitley; Paige Kaplan; Stuart J Swiedler; Susan Conrad; Paul Harmatz
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Publication Detail:
Type:  JOURNAL ARTICLE    
Journal Detail:
Title:  Journal of pediatric rehabilitation medicine     Volume:  3     ISSN:  1875-8894     ISO Abbreviation:  -     Publication Date:  2010  
Date Detail:
Created Date:  2010-7-16     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101490944     Medline TA:  J Pediatr Rehabil Med     Country:  -    
Other Details:
Languages:  ENG     Pagination:  89-100     Citation Subset:  -    
Affiliation:
BioMarin Pharmaceutical Inc, Novato, CA, USA.
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MeSH Terms
Descriptor/Qualifier:
Grant Support
ID/Acronym/Agency:
M01 RR001271-21//NCRR NIH HHS

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