For Publishers
DiscoveryMetadataPeer reviewHostingPublishing
For Researchers
JoinPublishReviewCollect
Register
Blog
About
Search
Advanced search
My ScienceOpen
Search
For Publishers
For Researchers
Blog
About
5
views
33
references
 Top references
cited by
1
    
0 reviews
 Review
0
comments
 Comment
0
recommends
+1 Recommend
0 collections
    0
    shares
      203
      similar
       All similar
      • Record: found
      • Abstract: found
      • Article: found
      Is Open Access

      Exploring the relationship between North Star Ambulatory Assessment and Health Utilities Index scores in Duchenne muscular dystrophy

      research-article

      Read this article at

      ScienceOpenPublisherPMC
      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          Abstract

          Background

          The North Star Ambulatory Assessment (NSAA) documents motor performance in ambulatory individuals with Duchenne muscular dystrophy (DMD). Health Utilities Index (HUI) scores, reflecting preferences for health-related quality-of-life (HRQoL) implications of health states, are commonly estimated within trials. This study sought to characterize the relationship between the NSAA score and utility in DMD.

          Methods

          Family members serving as proxy respondents for placebo-treated ambulatory individuals with DMD (NCT01254019; BioMarin Pharmaceuticals Inc) completed the HUI and the NSAA (score range, 0–34). Mean change over time on these measures was estimated, and the correlation between changes in NSAA score and a) HUI utility; b) HUI3 ambulation and HUI2 mobility attribute scores, over 48 weeks was calculated.

          Results

          Baseline mean (range) age was 8.0 years (5–16; n = 61) and mean (standard deviation [SD]) scores were 0.87 (0.13; HUI2), 0.82 (0.19; HUI3), and 21.0 (8.1; NSAA). Mean (SD) change over 48 weeks was –0.05 (0.14; HUI2), –0.06 (0.19; HUI3), and –2.9 (4.7; NSAA). Weak positive correlations were observed between baseline NSAA score and HUI utility (HUI2: r = 0.29; HUI3: r = 0.17) and for change over 48 weeks (HUI2: r = 0.16; HUI3: r = 0.15). Stronger correlations were observed between change in NSAA score and the HUI3 ambulation ( r = 0.41) and HUI2 mobility ( r = 0.41) attributes.

          Conclusions

          Among ambulatory individuals with DMD, NSAA score is weakly correlated with HUI utility, suggesting that motor performance alone does not fully explain HRQoL. Stronger relationships were observed between HUI ambulation and mobility attributes, and NSAA. Although unidimensional measures like the NSAA are informative for documenting disease-specific health impacts, they may not correlate well with measures of overall health status; requiring use in conjunction with other patient-reported and preference-based outcomes.

          Supplementary Information

          The online version contains supplementary material available at 10.1186/s12955-023-02160-8.

          Related collections

          Most cited references33

          • Record: found
          • Abstract: found
          • Article: not found

          The Health Utilities Index (HUI®): concepts, measurement properties and applications

          John Horsman, William Furlong, David H. Feeny … (2003)
          This is a review of the Health Utilities Index (HUI®) multi-attribute health-status classification systems, and single- and multi-attribute utility scoring systems. HUI refers to both HUI Mark 2 (HUI2) and HUI Mark 3 (HUI3) instruments. The classification systems provide compact but comprehensive frameworks within which to describe health status. The multi-attribute utility functions provide all the information required to calculate single-summary scores of health-related quality of life (HRQL) for each health state defined by the classification systems. The use of HUI in clinical studies for a wide variety of conditions in a large number of countries is illustrated. HUI provides comprehensive, reliable, responsive and valid measures of health status and HRQL for subjects in clinical studies. Utility scores of overall HRQL for patients are also used in cost-utility and cost-effectiveness analyses. Population norm data are available from numerous large general population surveys. The widespread use of HUI facilitates the interpretation of results and permits comparisons of disease and treatment outcomes, and comparisons of long-term sequelae at the local, national and international levels.
          Article 0 comments Cited 141 times – based on 0 reviews     Review now
            Bookmark
            • Record: found
            • Abstract: found
            • Article: not found

            Duchenne muscular dystrophy.

            Eppie M. Yiu, Andrew J. Kornberg (2015)
            Duchenne muscular dystrophy, an X-linked disorder, has an incidence of one in 5000 boys and presents in early childhood with proximal muscle weakness. Untreated boys become wheelchair bound by the age of 12 years and die of cardiorespiratory complications in their late teens to early 20s. The use of corticosteroids, non-invasive respiratory support, and active surveillance and management of associated complications have improved ambulation, function, quality of life and life expectancy. The clinical features, investigations and management of Duchenne muscular dystrophy are reviewed, as well as the latest in some of the novel therapies.
            Article 0 comments Cited 90 times – based on 0 reviews     Review now
              Bookmark
              • Record: found
              • Abstract: found
              • Article: not found

              North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy.

              Chiara De Mutiis, Enrico Bertini, Angela Berardinelli … (2010)
              The North Star Ambulatory Assessment is a functional scale specifically designed for ambulant boys affected by Duchenne muscular dystrophy (DMD). Recently the 6-minute walk test has also been used as an outcome measure in trials in DMD. The aim of our study was to assess a large cohort of ambulant boys affected by DMD using both North Star Assessment and 6-minute walk test. More specifically, we wished to establish the spectrum of findings for each measure and their correlation. This is a prospective multicentric study involving 10 centers. The cohort included 112 ambulant DMD boys of age ranging between 4.10 and 17 years (mean 8.18±2.3 DS). Ninety-one of the 112 were on steroids: 37/91 on intermittent and 54/91 on daily regimen. The scores on the North Star assessment ranged from 6/34 to 34/34. The distance on the 6-minute walk test ranged from 127 to 560.6 m. The time to walk 10 m was between 3 and 15 s. The time to rise from the floor ranged from 1 to 27.5 s. Some patients were unable to rise from the floor. As expected the results changed with age and were overall better in children treated with daily steroids. The North Star assessment had a moderate to good correlation with 6-minute walk test and with timed rising from floor but less with 10 m timed walk/run test. The 6-minute walk test in contrast had better correlation with 10 m timed walk/run test than with timed rising from floor. These findings suggest that a combination of these outcome measures can be effectively used in ambulant DMD boys and will provide information on different aspects of motor function, that may not be captured using a single measure. Copyright © 2010. Published by Elsevier B.V.
              Article 0 comments Cited 72 times – based on 0 reviews     Review now
                Bookmark
                All references

                Author and article information

                Contributors
                Shelagh M. Szabo:
                ORCID: http://orcid.org/0000-0002-9044-3192
                sszabo@broadstreetheor.com
                Journal
                Journal ID (nlm-ta): Health Qual Life Outcomes
                Journal ID (iso-abbrev): Health Qual Life Outcomes
                Title: Health and Quality of Life Outcomes
                Publisher: BioMed Central (London )
                ISSN (Electronic): 1477-7525
                Publication date (Electronic): 19 July 2023
                Publication date PMC-release: 19 July 2023
                Publication date Collection: 2023
                Volume: 21
                Electronic Location Identifier: 76
                Affiliations
                [1 ]GRID grid.423097.b, ISNI 0000 0004 0408 3130, Sarepta Therapeutics, Inc., ; Cambridge, MA USA
                [2 ]Broadstreet HEOR, 201 – 343 Railway St, Vancouver, BC V6A 1A6 Canada
                [3 ]GRID grid.25073.33, ISNI 0000 0004 1936 8227, McMaster University and Health Utilities Inc, ; Hamilton, ON Canada
                Author information
                http://orcid.org/0000-0002-9044-3192
                Article
                Publisher ID: 2160
                DOI: 10.1186/s12955-023-02160-8
                PMC ID: 10355009
                PubMed ID: 37468890
                SO-VID: bef444b4-1269-4dd8-8992-776be7a43165
                Copyright © © The Author(s) 2023
                License:

                Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver ( http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.

                History
                Date received : 31 March 2023
                Date accepted : 7 July 2023
                Funding
                Funded by: FundRef http://dx.doi.org/10.13039/100014943, Sarepta Therapeutics;
                Categories
                Subject: Research
                Custom metadata
                issue-copyright-statement © BioMed Central Ltd., part of Springer Nature 2023

                ScienceOpen disciplines: Health & Social care
                Keywords: duchenne muscular dystrophy,utility,nsaa,hui
                Data availability:
                ScienceOpen disciplines: Health & Social care
                Keywords: duchenne muscular dystrophy, utility, nsaa, hui

                Comments

                Comment on this article

                scite_

                Similar content203

                See all similar

                Cited by1

                See all cited by

                Most referenced authors437

                See all reference authors