{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T16:09:38Z","timestamp":1753891778074,"version":"3.41.2"},"reference-count":36,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T00:00:00Z","timestamp":1747094400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurol."],"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>The Patient-Reported Outcomes Measurement Information System (PROMIS) is a collection of generic patient-reported outcome instruments used to quantify disease impact on a variety of functional subdomains, including physical, cognitive, emotional, and other domains. The reliability and validity of the PROMIS Parent Proxy (PP) Physical Function\u2013Upper Extremity (UE) item bank is not established in children with Duchenne muscular dystrophy (DMD). This study evaluated the psychometric properties and measurement quality of the PROMIS PP UE item bank v1.0 (29 items) in DMD using a Rasch psychometric analysis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The PROMIS PP UE item bank was completed by caregivers of children with DMD aged at least 8\u202fyears, under care at Nationwide Children\u2019s Hospital (Columbus, OH, United States). Rasch analysis was used to evaluate the psychometric performance of the measure and its items in DMD, based on several criteria, including item\u2013trait interaction, individual items fit, Person Separation Index (PSI), individual persons fit, and response dependency.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Rasch analysis was conducted on 206 observations. Several items had weak clinical utility in measuring upper extremity functioning in DMD. Additionally, the analysis identified specific response options that could be restructured to improve the reliability and precision of the items in evaluating upper extremity function in DMD. A new customized 21-item measure demonstrated overall good fit to Rasch model expectations (<jats:italic>p<\/jats:italic>\u202f=\u202f0.095; nonsignificant) and the ability to discriminate among respondents with different levels of upper extremity function (0.95 PSI; excellent reliability). Upper extremity function was generally well targeted across the severity spectrum, except for the least severe patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The customized PROMIS PP UE measure conformed to Rasch assumptions, indicating that it can serve as a reliable option for caregiver-reported upper extremity assessment in DMD.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fneur.2025.1481825","type":"journal-article","created":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T16:34:46Z","timestamp":1747154086000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Psychometric analysis of the patient-reported outcomes measurement information system parent proxy physical function\u2013upper extremity item bank for children with Duchenne muscular dystrophy"],"prefix":"10.3389","volume":"16","author":[{"given":"Linda Pax","family":"Lowes","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Corinne M.","family":"Le Reun","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Teofil","family":"Ciobanu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Natalie F.","family":"Reash","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Megan A.","family":"Iammarino","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shivangi","family":"Patel","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ivana F.","family":"Audhya","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2025,5,13]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1186\/s13023-021-01862-w","article-title":"The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review","volume":"16","author":"Szabo","year":"2021","journal-title":"Orphanet J Rare Dis"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"593","DOI":"10.1007\/s11136-019-02355-x","article-title":"Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review","volume":"29","author":"Szabo","year":"2020","journal-title":"Qual Life Res"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1186\/s12955-020-01511-z","article-title":"Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments","volume":"18","author":"Powell","year":"2020","journal-title":"Health Qual Life Outcomes"},{"key":"ref4","doi-asserted-by":"publisher","first-page":"141","DOI":"10.1186\/s13023-020-01430-8","article-title":"Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis","volume":"15","author":"Crisafulli","year":"2020","journal-title":"Orphanet J Rare Dis"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"1810","DOI":"10.1007\/s00415-016-8193-1","article-title":"Variables associated with upper extremity function in patients with Duchenne muscular dystrophy","volume":"263","author":"Janssen","year":"2016","journal-title":"J Neurol"},{"key":"ref6","doi-asserted-by":"publisher","first-page":"469","DOI":"10.3233\/jnd-200548","article-title":"Comparison of long-term ambulatory function in patients with Duchenne muscular dystrophy treated with eteplirsen and matched natural history controls","volume":"8","author":"Mendell","year":"2021","journal-title":"J Neuromuscul Dis"},{"key":"ref7","doi-asserted-by":"publisher","first-page":"79","DOI":"10.1186\/s13023-017-0631-3","article-title":"The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review","volume":"12","author":"Ryder","year":"2017","journal-title":"Orphanet J Rare Dis"},{"key":"ref8","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1186\/s41687-023-00669-6","article-title":"A qualitative study to understand the Duchenne muscular dystrophy experience from the parent\/patient perspective","volume":"7","author":"Brown","year":"2023","journal-title":"J Patient Rep Outcomes"},{"key":"ref9","doi-asserted-by":"publisher","first-page":"224","DOI":"10.1111\/dmcn.13277","article-title":"Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD upper limb PROM","volume":"59","author":"Klingels","year":"2017","journal-title":"Dev Med Child Neurol"},{"key":"ref10","doi-asserted-by":"publisher","first-page":"61","DOI":"10.1186\/s13023-018-0810-x","article-title":"Patient reported outcome measures in rare diseases: a narrative review","volume":"13","author":"Slade","year":"2018","journal-title":"Orphanet J Rare Dis"},{"key":"ref11","doi-asserted-by":"publisher","first-page":"487","DOI":"10.1186\/s13023-021-02114-7","article-title":"Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks","volume":"16","author":"Schwartz","year":"2021","journal-title":"Orphanet J Rare Dis"},{"key":"ref12","doi-asserted-by":"publisher","first-page":"g7818","DOI":"10.1136\/bmj.g7818","article-title":"Patient reported outcome measures in practice","volume":"350","author":"Nelson","year":"2015","journal-title":"BMJ"},{"key":"ref13","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1186\/s12955-018-1062-0","article-title":"A review of quality of life themes in Duchenne muscular dystrophy for patients and carers","volume":"16","author":"Uttley","year":"2018","journal-title":"Health Qual Life Outcomes"},{"key":"ref14","doi-asserted-by":"publisher","first-page":"e2438","DOI":"10.1212\/wnl.0000000000011896","article-title":"Development of a new quality of life measure for Duchenne muscular dystrophy using mixed methods: the DMD-QoL","volume":"96","author":"Powell","year":"2021","journal-title":"Neurology"},{"key":"ref15","doi-asserted-by":"publisher","first-page":"1499","DOI":"10.1016\/j.jval.2021.03.007","article-title":"Deriving a preference-based measure for people with Duchenne muscular dystrophy from the DMD-QoL","volume":"24","author":"Rowen","year":"2021","journal-title":"Value Health"},{"key":"ref16","doi-asserted-by":"publisher","first-page":"e1559","DOI":"10.1542\/peds.2012-0858","article-title":"Health-related quality of life in children and adolescents with Duchenne muscular dystrophy","volume":"130","author":"Uzark","year":"2012","journal-title":"Pediatrics"},{"key":"ref17","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1186\/s41687-022-00425-2","article-title":"Interplay of disability, caregiver impact, and out-of-pocket expenditures in Duchenne muscular dystrophy: a cohort study","volume":"6","author":"Schwartz","year":"2022","journal-title":"J Patient Rep Outcomes"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.16198","article-title":"Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy","author":"Lowes","year":"2024","journal-title":"Dev Med Child Neurol"},{"key":"ref19","doi-asserted-by":"publisher","first-page":"332","DOI":"10.1038\/s41591-024-03304-z","article-title":"AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial","volume":"31","author":"Mendell","year":"2024","journal-title":"Nat Med"},{"key":"ref20","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1007\/s12402-016-0210-9","article-title":"Is there a difference between child self-ratings and parent proxy-ratings of the quality of life of children with a diagnosis of attention-deficit hyperactivity disorder (ADHD)? A systematic review of the literature","volume":"9","author":"Galloway","year":"2017","journal-title":"Attention Defic. Hyperact. Dis."},{"key":"ref21","doi-asserted-by":"publisher","first-page":"45","DOI":"10.1186\/s12955-021-01677-0","article-title":"A comparison of self-reported and proxy-reported health utilities in children: a systematic review and meta-analysis","volume":"19","author":"Jiang","year":"2021","journal-title":"Health Qual Life Outcomes"},{"key":"ref22","doi-asserted-by":"publisher","first-page":"R144","DOI":"10.1186\/ar4327","article-title":"Development and assessment of floor and ceiling items for the PROMIS physical function item bank","volume":"15","author":"Bruce","year":"2013","journal-title":"Arthritis Res Ther"},{"key":"ref23","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/B978-0-12-801504-9.00010-6","article-title":"Chapter 10\u2014item response theory","volume-title":"The measurement of health and health status","author":"Krabbe","year":"2017"},{"key":"ref24","doi-asserted-by":"publisher","first-page":"149","DOI":"10.1007\/BF02296272","article-title":"A Rasch model for partial credit scoring","volume":"47","author":"Masters","year":"1982","journal-title":"Psychometrika"},{"key":"ref25","first-page":"205","article-title":"Detecting and evaluating the impact of multidimensionality using item fit statistics and principal component analysis of residuals","volume":"3","author":"Smith","year":"2002","journal-title":"J Appl Meas"},{"key":"ref26","first-page":"1048","article-title":"Unidimensionality matters! (a tale of two Smiths?)","volume":"20","author":"Tennant","year":"2006","journal-title":"Rasch Meas Trans"},{"key":"ref27","doi-asserted-by":"publisher","first-page":"1358","DOI":"10.1002\/art.23108","article-title":"The Rasch measurement model in rheumatology: what is it and why use it? When should it be applied, and what should one look for in a Rasch paper?","volume":"57","author":"Tennant","year":"2007","journal-title":"Arthritis Rheum"},{"key":"ref28","doi-asserted-by":"publisher","first-page":"69","DOI":"10.1016\/j.nmd.2023.07.003","article-title":"Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0","volume":"33","author":"Cicala","year":"2023","journal-title":"Neuromuscul Disord"},{"key":"ref29","doi-asserted-by":"publisher","first-page":"459","DOI":"10.1016\/j.nmd.2007.02.013","article-title":"ACTIVLIM: a Rasch-built measure of activity limitations in children and adults with neuromuscular disorders","volume":"17","author":"Vandervelde","year":"2007","journal-title":"Neuromuscul Disord"},{"key":"ref30","doi-asserted-by":"publisher","first-page":"114","DOI":"10.1186\/1471-2474-9-114","article-title":"The disabilities of the arm, shoulder and hand questionnaire (DASH) can measure the impairment, activity limitations and participation restriction constructs from the international classification of functioning, disability and health (ICF)","volume":"9","author":"Dixon","year":"2008","journal-title":"BMC Musculoskelet Disord"},{"key":"ref31","doi-asserted-by":"publisher","first-page":"2448","DOI":"10.1080\/09638288.2020.1829107","article-title":"Reliability and validity of upper limb short questionnaire for Duchenne muscular dystrophy","volume":"44","author":"Choi","year":"2022","journal-title":"Disabil Rehabil"},{"key":"ref32","doi-asserted-by":"publisher","first-page":"1179","DOI":"10.1016\/j.jclinepi.2010.04.011","article-title":"The patient-reported outcomes measurement information system (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008","volume":"63","author":"Cella","year":"2010","journal-title":"J Clin Epidemiol"},{"key":"ref33","doi-asserted-by":"publisher","first-page":"69","DOI":"10.1186\/s41687-019-0158-6","article-title":"The expansion and validation of a new upper extremity item bank for the patient-reported outcomes measurement information system\u00ae (PROMIS)","volume":"3","author":"Kaat","year":"2019","journal-title":"J Patient Rep Outcomes"},{"key":"ref34","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1016\/j.jclinepi.2015.08.038","article-title":"PROMIS measures of pain, fatigue, negative affect, physical function, and social function demonstrated clinical validity across a range of chronic conditions","volume":"73","author":"Cook","year":"2016","journal-title":"J Clin Epidemiol"},{"key":"ref35","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1002\/cncr.30354","article-title":"Responsiveness of 8 patient-reported outcomes measurement information system (PROMIS) measures in a large, community-based cancer study cohort","volume":"123","author":"Jensen","year":"2017","journal-title":"Cancer"},{"key":"ref36","doi-asserted-by":"publisher","first-page":"485","DOI":"10.1007\/s40271-020-00493-w","article-title":"The use of patient-reported outcome measures in rare diseases and implications for health technology assessment","volume":"14","author":"Whittal","year":"2021","journal-title":"Patient"}],"container-title":["Frontiers in Neurology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2025.1481825\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,5,26]],"date-time":"2025-05-26T08:54:22Z","timestamp":1748249662000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2025.1481825\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,5,13]]},"references-count":36,"alternative-id":["10.3389\/fneur.2025.1481825"],"URL":"https:\/\/doi.org\/10.3389\/fneur.2025.1481825","relation":{},"ISSN":["1664-2295"],"issn-type":[{"type":"electronic","value":"1664-2295"}],"subject":[],"published":{"date-parts":[[2025,5,13]]},"article-number":"1481825"}}