Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome
J. inborn errors metab. screen
;
3: e140016, 2015. tab, graf
Article
in English
|
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.
Full text:
Available
Index:
LILACS (Americas)
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2015
Type:
Article
Affiliation country:
Belgium
/
Germany
/
United States
/
United kingdom
Institution/Affiliation country:
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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