Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome
Autor(a) principal: | |
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Data de Publicação: | 2015 |
Outros Autores: | , , , , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Journal of Inborn Errors of Metabolism and Screening |
Texto Completo: | http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942015000100802 |
Resumo: | 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. |
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Journal of Inborn Errors of Metabolism and Screening |
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Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A SyndromeMorquio Amucopolysaccharidosis IVpatient-reported outcomesquality of lifeEQ5D-5Lpatient’s perception6MWT3MSC testFVCmaximal voluntary ventilationmobilityphysical endurancerespiratory function testsAbstract 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.Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT)2015-01-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942015000100802Journal of Inborn Errors of Metabolism and Screening v.3 2015reponame:Journal of Inborn Errors of Metabolism and Screeninginstname:Instituto Genética para Todos (IGPT)instacron:IGPT10.1177/2326409815576188info:eu-repo/semantics/openAccessLampe,ChristinaJain,MohitOlaye,AndrewMeesen,BiancaDecker,CelesteMengel,Eugeneng2019-06-17T00:00:00Zoai:scielo:S2326-45942015000100802Revistahttp://jiems-journal.org/ONGhttps://old.scielo.br/oai/scielo-oai.phpjiems@jiems-journal.org||rgiugliani@hcpa.edu.br2326-45942326-4594opendoar:2019-06-17T00:00Journal of Inborn Errors of Metabolism and Screening - Instituto Genética para Todos (IGPT)false |
dc.title.none.fl_str_mv |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
title |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
spellingShingle |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome Lampe,Christina Morquio A mucopolysaccharidosis IV patient-reported outcomes quality of life EQ5D-5L patient’s perception 6MWT 3MSC test FVC maximal voluntary ventilation mobility physical endurance respiratory function tests |
title_short |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
title_full |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
title_fullStr |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
title_full_unstemmed |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
title_sort |
Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome |
author |
Lampe,Christina |
author_facet |
Lampe,Christina Jain,Mohit Olaye,Andrew Meesen,Bianca Decker,Celeste Mengel,Eugen |
author_role |
author |
author2 |
Jain,Mohit Olaye,Andrew Meesen,Bianca Decker,Celeste Mengel,Eugen |
author2_role |
author author author author author |
dc.contributor.author.fl_str_mv |
Lampe,Christina Jain,Mohit Olaye,Andrew Meesen,Bianca Decker,Celeste Mengel,Eugen |
dc.subject.por.fl_str_mv |
Morquio A mucopolysaccharidosis IV patient-reported outcomes quality of life EQ5D-5L patient’s perception 6MWT 3MSC test FVC maximal voluntary ventilation mobility physical endurance respiratory function tests |
topic |
Morquio A mucopolysaccharidosis IV patient-reported outcomes quality of life EQ5D-5L patient’s perception 6MWT 3MSC test FVC maximal voluntary ventilation mobility physical endurance respiratory function tests |
description |
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. |
publishDate |
2015 |
dc.date.none.fl_str_mv |
2015-01-01 |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942015000100802 |
url |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942015000100802 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
10.1177/2326409815576188 |
dc.rights.driver.fl_str_mv |
info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
text/html |
dc.publisher.none.fl_str_mv |
Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT) |
publisher.none.fl_str_mv |
Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT) |
dc.source.none.fl_str_mv |
Journal of Inborn Errors of Metabolism and Screening v.3 2015 reponame:Journal of Inborn Errors of Metabolism and Screening instname:Instituto Genética para Todos (IGPT) instacron:IGPT |
instname_str |
Instituto Genética para Todos (IGPT) |
instacron_str |
IGPT |
institution |
IGPT |
reponame_str |
Journal of Inborn Errors of Metabolism and Screening |
collection |
Journal of Inborn Errors of Metabolism and Screening |
repository.name.fl_str_mv |
Journal of Inborn Errors of Metabolism and Screening - Instituto Genética para Todos (IGPT) |
repository.mail.fl_str_mv |
jiems@jiems-journal.org||rgiugliani@hcpa.edu.br |
_version_ |
1754732519901626368 |