Physical Function and Health-Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric-Onset Hypophosphatasia.
CLINICAL STUDY
ENZYME REPLACEMENT THERAPY
HYPOPHOSPHATASIA
PHYSICAL PERFORMANCE
REAL‐WORLD EVIDENCE
Journal
JBMR plus
ISSN: 2473-4039
Titre abrégé: JBMR Plus
Pays: England
ID NLM: 101707013
Informations de publication
Date de publication:
Sep 2020
Sep 2020
Historique:
received:
07
04
2020
revised:
02
07
2020
accepted:
13
07
2020
entrez:
30
9
2020
pubmed:
1
10
2020
medline:
1
10
2020
Statut:
epublish
Résumé
Hypophosphatasia (HPP) is a rare, inherited, metabolic disease characterized by tissue-nonspecific alkaline phosphatase deficiency resulting in musculoskeletal and systemic clinical manifestations. This observational study evaluated the effectiveness of enzyme replacement therapy with asfotase alfa on physical function and health-related quality of life (HRQoL) among adults with pediatric-onset HPP who received asfotase alfa for 12 months at a single center (ClinicalTrial.gov no.: NCT03418389). Primary outcomes evaluated physical function with the 6-minute walk test (6MWT), timed up-and-go (TUG) test, Short Physical Performance Battery (SPPB), and handheld dynamometry (HHD). Secondary outcome measures included the Lower Extremity Functional Scale (LEFS), pain prevalence/intensity, and pain medication use; HRQoL was evaluated using the 36-Item Short-Form Health Survey version 2 (SF-36v2). Safety data were collected throughout the study. All 14 patients (11 women) had compound heterozygous
Identifiants
pubmed: 32995696
doi: 10.1002/jbm4.10395
pii: JBM410395
pmc: PMC7507107
doi:
Types de publication
Clinical Trial
Langues
eng
Pagination
e10395Informations de copyright
© 2020 The Authors. JBMR Plus published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research.
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