أعرض في EDS

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

التفاصيل البيبلوغرافية
العنوان: Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study
المؤلفون: Strive Investigators, Wolfgang Dummer, Simon Jones, Roberto Giugliani, Maurizio Scarpa, Vassili Valayannopoulos, Shuan-Pei Lin, Paul Harmatz, Debra L. Lounsbury, Barbara K. Burton, Peter Slasor, Thomas R. Fleming, Derralynn Hughes, Eugen Mengel, Christian J. Hendriksz
المصدر: STRIVE Investigators 2014, ' Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA) : A phase 3 randomised placebo-controlled study ', Journal of Inherited Metabolic Disease, vol. 37, no. 6, pp. 979-990 . https://doi.org/10.1007/s10545-014-9715-6
Journal of Inherited Metabolic Disease
بيانات النشر: Wiley, 2014.
سنة النشر: 2014
مصطلحات موضوعية: Adult, Male, congenital, hereditary, and neonatal diseases and abnormalities, medicine.medical_specialty, Morquio syndrome, Adolescent, Placebo-controlled study, Walking, Motor Activity, Placebo, law.invention, Efficacy, Young Adult, chemistry.chemical_compound, Rare Diseases, Double-Blind Method, Randomized controlled trial, Elosulfase alfa, law, Internal medicine, Genetics, medicine, Humans, Enzyme Replacement Therapy, Genetics(clinical), Respiratory function, Child, Genetics (clinical), business.industry, fungi, Mucopolysaccharidosis IV, virus diseases, nutritional and metabolic diseases, Middle Aged, medicine.disease, Chondroitinsulfatases, 3. Good health, Discontinuation, Treatment Outcome, chemistry, Keratan Sulfate, Child, Preschool, Original Article, Female, business
الوصف: Objective To assess the efficacy and safety of enzyme replacement therapy (ERT) with BMN 110 (elosulfase alfa) in patients with Morquio A syndrome (mucopolysaccharidosis IVA). Methods Patients with Morquio A aged ≥5 years (N = 176) were randomised (1:1:1) to receive elosulfase alfa 2.0 mg/kg/every other week (qow), elosulfase alfa 2.0 mg/kg/week (weekly) or placebo for 24 weeks in this phase 3, double-blind, randomised study. The primary efficacy measure was 6-min walk test (6MWT) distance. Secondary efficacy measures were 3-min stair climb test (3MSCT) followed by change in urine keratan sulfate (KS). Various exploratory measures included respiratory function tests. Patient safety was also evaluated. Results At week 24, the estimated mean effect on the 6MWT versus placebo was 22.5 m (95 % CI 4.0, 40.9; P = 0.017) for weekly and 0.5 m (95 % CI −17.8, 18.9; P = 0.954) for qow. The estimated mean effect on 3MSCT was 1.1 stairs/min (95 % CI −2.1, 4.4; P = 0.494) for weekly and −0.5 stairs/min (95 % CI −3.7, 2.8; P = 0.778) for qow. Normalised urine KS was reduced at 24 weeks in both regimens. In the weekly dose group, 22.4 % of patients had adverse events leading to an infusion interruption/discontinuation requiring medical intervention (only 1.3 % of all infusions in this group) over 6 months. No adverse events led to permanent treatment discontinuation. Conclusions Elosulfase alfa improved endurance as measured by the 6MWT in the weekly but not qow dose group, did not improve endurance on the 3MSCT, reduced urine KS, and had an acceptable safety profile. Electronic supplementary material The online version of this article (doi:10.1007/s10545-014-9715-6) contains supplementary material, which is available to authorized users.
تدمد: 1573-2665
0141-8955
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3ce90913ba268a2a462fbbf7743596e0
https://doi.org/10.1007/s10545-014-9715-6
حقوق: OPEN
رقم الأكسشن: edsair.doi.dedup.....3ce90913ba268a2a462fbbf7743596e0
قاعدة البيانات: OpenAIRE
الوصف
تدمد:15732665
01418955