{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T04:19:08Z","timestamp":1784607548032,"version":"3.55.0"},"reference-count":21,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2016,9,26]],"date-time":"2016-09-26T00:00:00Z","timestamp":1474848000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100007423","name":"Duchenne Parent Project, the Netherlands","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100007423","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2017,2]]},"abstract":"<jats:sec><jats:title>Aim<\/jats:title><jats:p>To develop a patient\u2010reported outcome measure (<jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content>) assessing upper limb function related to activities of daily living (<jats:styled-content style=\"fixed-case\">ADL<\/jats:styled-content>) that cannot be observed in a clinical setting, specifically for patients with Duchenne muscular dystrophy (<jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content>) across a wide age range, applicable in the different stages of the disease.<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>The developmental process was based on <jats:styled-content style=\"fixed-case\">US<\/jats:styled-content> Food and Drug Administration guidelines. This included item generation from a systematic review of existing tools and expert opinion on task difficulty and relevance, involving individuals with <jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content>. Cultural aspects affecting <jats:styled-content style=\"fixed-case\">ADL<\/jats:styled-content> were taken into consideration to make this tool applicable to the broad <jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content> community. Items were selected in relation to a conceptual framework reflecting disease progression covering the full range of upper limb function across different <jats:styled-content style=\"fixed-case\">ADL<\/jats:styled-content> domains.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>After pilot testing and iterative Rasch analyses, redundant or clinically irrelevant items were removed. The final questionnaire consists of 32 items covering four domains of <jats:styled-content style=\"fixed-case\">ADL<\/jats:styled-content> (food, self\u2010care, household and environment, leisure and communication). Test\u2013retest reliability was excellent.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>A <jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content>\u2010specific upper limb <jats:styled-content style=\"fixed-case\">PROM<\/jats:styled-content> was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self\u2010reported natural history of <jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content> and assess the efficacy of interventions.<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.13277","type":"journal-article","created":{"date-parts":[[2016,9,27]],"date-time":"2016-09-27T04:03:36Z","timestamp":1474949016000},"page":"224-231","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":44,"title":["Development of a patient\u2010reported outcome measure for upper limb function in Duchenne muscular dystrophy: <scp>DMD<\/scp> Upper Limb <scp>PROM<\/scp>"],"prefix":"10.1111","volume":"59","author":[{"given":"K","family":"Klingels","sequence":"first","affiliation":[{"name":"Department of Rehabilitation Sciences KU Leuven Leuven Belgium"},{"name":"Rehabilitation Research Center (REVAL) Biomed Hasselt University Diepenbeek Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A G","family":"Mayhew","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre Institute of Genetic Medicine Newcastle upon Tyne UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E S","family":"Mazzone","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology Catholic University Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T","family":"Duong","sequence":"additional","affiliation":[{"name":"Department of Neurology Stanford University School of Medicine Stanford CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V","family":"Decostre","sequence":"additional","affiliation":[{"name":"Institut de Myologie GH Piti\u00e9 Salp\u00eatri\u00e8re Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"U","family":"Werlauff","sequence":"additional","affiliation":[{"name":"Danish National Rehabilitation Center for Neuromuscular Diseases Aarhus Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E","family":"Vroom","sequence":"additional","affiliation":[{"name":"Duchenne Parent Project Veenendaal the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology Catholic University Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"N M","family":"Goemans","sequence":"additional","affiliation":[{"name":"Department of Child Neurology University Hospitals Leuven Leuven Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"The Upper Limb Clinical Outcome Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,9,26]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.06.014"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2011.03939.x"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21808"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0113999"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-014-7316-9"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2012.06.006"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.12213"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.11.014"},{"key":"e_1_2_7_10_1","volume-title":"Guidance for Industry. 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