Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome
J. inborn errors metab. screen
;
3: e140016, 2015. tab, graf
Artigo
em Inglês
|
LILACS-Express
| LILACS
| ID: biblio-1090870
ABSTRACT
Abstract This cross-sectional analysis assessed the correlation between patient-reported outcomes (PROs) and clinical outcomes in 24 German patients with Morquio A. Clinical outcomes included 6-minute walk test (6MWT), 3-minute stair climb (3MSC) test, and joint range of motion as measures for endurance/mobility, forced vital capacity (FVC) and maximum voluntary ventilation (MVV) as measures for respiratory function, and height as an important manifestation. The PROs included the EuroQoL (EQ) 5D-5L (EQ5D-5L), to measure health-related QoL (HRQoL), and patients' rating of their ability to walk, climb, or breathe. In adults, endurance and pulmonary function measures and height showed strong and statistically significant correlation with the patients' EQ5D-5L (6MWT R = .884, 3MSC test R = .852, FVC R = .815, MVV R = .825, height R = .842). The adult patients' rating of their ability to walk and climb also correlated strongly with 6MWT (R = .839) and 3MSC test (R = .700) results. Improvements in these clinical outcomes may be robust surrogate parameters of a better EQ5D-5L/HRQoL in patients with Morquio A.
Texto completo:
DisponíveL
Índice:
LILACS (Américas)
Idioma:
Inglês
Revista:
J. inborn errors metab. screen
Assunto da revista:
Medicina Cl¡nica
/
Patologia
Ano de publicação:
2015
Tipo de documento:
Artigo
País de afiliação:
Bélgica
/
Alemanha
/
Estados Unidos
/
Reino Unido
Instituição/País de afiliação:
BioMarin Europe Ltd/GB
/
BioMarin Pharmaceutical Inc/US
/
Clinic for children and adolescents/DE
/
Ismar Healthcare/BE
/
Johannes Gutenberg-University/DE
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