A. Pearlmutter , D. Powell , D. Carlton , E. Chen , K. Gallington , D. Bacci , D. Gelhorn , M. Cho , D. Dashiell-Aje
{"title":"167p对4-7岁杜氏肌营养不良症患儿的照顾者进行定性访谈,评估代理填写的DMD-QOL的内容效度","authors":"A. Pearlmutter , D. Powell , D. Carlton , E. Chen , K. Gallington , D. Bacci , D. Gelhorn , M. Cho , D. Dashiell-Aje","doi":"10.1016/j.nmd.2025.105520","DOIUrl":null,"url":null,"abstract":"<div><div>The Duchenne muscular dystrophy quality of life (DMD-QoL) is a 14-item questionnaire designed to assess physical functioning, psychological impact, and social participation among males with Duchenne muscular dystrophy (DMD) that has been validated for use in boys aged 7+ years by proxy report and boys aged 10+ years by self-report. There is currently an evidence gap for use of the proxy-completed DMD-QoL among younger DMD patients. We conducted qualitative concept elicitation/cognitive interviews with caregivers of children under 7 years of age with DMD in the UK and US. The interviews explored the patient experience with DMD and assessed the relevance, comprehensiveness, ease of use, and understandability of the proxy-completed DMD-QoL. Interviews were completed with 7 caregivers (all parents) of children aged 5–7 years with DMD in the UK (n=3) and US (n=4); the mean age at DMD diagnosis was 3.4 years (range: 0–5.3 years). All caregivers (n=7/7) reported that the DMD-QoL instructions were understandable, the recall period was clear, and they were able to answer each item with the response options provided. Frequently endorsed concepts that were relevant for this age group included difficulty getting around (n=7/7), feeling tired (n=6/7), pain (n=6/7), and feeling unhappy (n=6/7). Fewer caregivers endorsed the relevance of psychological impact concepts (eg, he felt embarrassed [n=3/7), he found it hard to talk to people [n=3/7)) or reported they may not be easily attributable to DMD; however, most caregivers anticipated that such concepts would be relevant for their child in the future. Overall caregiver feedback indicated that the proxy-completed DMD-QoL is easy to use, understandable, and face valid for use by caregivers of children aged 4–7 years with DMD. A second phase of interviews should be conducted to further evaluate the measure’s generalizability and accessibility in this age group, including among children aged 4 years with DMD.</div></div>","PeriodicalId":19135,"journal":{"name":"Neuromuscular Disorders","volume":"53 ","pages":"Article 105520"},"PeriodicalIF":2.8000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"167PQualitative interviews with caregivers of children aged 4–7 years with Duchenne muscular dystrophy to assess content validity of the proxy-completed DMD-QOL\",\"authors\":\"A. Pearlmutter , D. Powell , D. Carlton , E. Chen , K. Gallington , D. Bacci , D. Gelhorn , M. Cho , D. Dashiell-Aje\",\"doi\":\"10.1016/j.nmd.2025.105520\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>The Duchenne muscular dystrophy quality of life (DMD-QoL) is a 14-item questionnaire designed to assess physical functioning, psychological impact, and social participation among males with Duchenne muscular dystrophy (DMD) that has been validated for use in boys aged 7+ years by proxy report and boys aged 10+ years by self-report. There is currently an evidence gap for use of the proxy-completed DMD-QoL among younger DMD patients. We conducted qualitative concept elicitation/cognitive interviews with caregivers of children under 7 years of age with DMD in the UK and US. The interviews explored the patient experience with DMD and assessed the relevance, comprehensiveness, ease of use, and understandability of the proxy-completed DMD-QoL. Interviews were completed with 7 caregivers (all parents) of children aged 5–7 years with DMD in the UK (n=3) and US (n=4); the mean age at DMD diagnosis was 3.4 years (range: 0–5.3 years). All caregivers (n=7/7) reported that the DMD-QoL instructions were understandable, the recall period was clear, and they were able to answer each item with the response options provided. Frequently endorsed concepts that were relevant for this age group included difficulty getting around (n=7/7), feeling tired (n=6/7), pain (n=6/7), and feeling unhappy (n=6/7). Fewer caregivers endorsed the relevance of psychological impact concepts (eg, he felt embarrassed [n=3/7), he found it hard to talk to people [n=3/7)) or reported they may not be easily attributable to DMD; however, most caregivers anticipated that such concepts would be relevant for their child in the future. Overall caregiver feedback indicated that the proxy-completed DMD-QoL is easy to use, understandable, and face valid for use by caregivers of children aged 4–7 years with DMD. A second phase of interviews should be conducted to further evaluate the measure’s generalizability and accessibility in this age group, including among children aged 4 years with DMD.</div></div>\",\"PeriodicalId\":19135,\"journal\":{\"name\":\"Neuromuscular Disorders\",\"volume\":\"53 \",\"pages\":\"Article 105520\"},\"PeriodicalIF\":2.8000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Neuromuscular Disorders\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0960896625002470\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neuromuscular Disorders","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0960896625002470","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
167PQualitative interviews with caregivers of children aged 4–7 years with Duchenne muscular dystrophy to assess content validity of the proxy-completed DMD-QOL
The Duchenne muscular dystrophy quality of life (DMD-QoL) is a 14-item questionnaire designed to assess physical functioning, psychological impact, and social participation among males with Duchenne muscular dystrophy (DMD) that has been validated for use in boys aged 7+ years by proxy report and boys aged 10+ years by self-report. There is currently an evidence gap for use of the proxy-completed DMD-QoL among younger DMD patients. We conducted qualitative concept elicitation/cognitive interviews with caregivers of children under 7 years of age with DMD in the UK and US. The interviews explored the patient experience with DMD and assessed the relevance, comprehensiveness, ease of use, and understandability of the proxy-completed DMD-QoL. Interviews were completed with 7 caregivers (all parents) of children aged 5–7 years with DMD in the UK (n=3) and US (n=4); the mean age at DMD diagnosis was 3.4 years (range: 0–5.3 years). All caregivers (n=7/7) reported that the DMD-QoL instructions were understandable, the recall period was clear, and they were able to answer each item with the response options provided. Frequently endorsed concepts that were relevant for this age group included difficulty getting around (n=7/7), feeling tired (n=6/7), pain (n=6/7), and feeling unhappy (n=6/7). Fewer caregivers endorsed the relevance of psychological impact concepts (eg, he felt embarrassed [n=3/7), he found it hard to talk to people [n=3/7)) or reported they may not be easily attributable to DMD; however, most caregivers anticipated that such concepts would be relevant for their child in the future. Overall caregiver feedback indicated that the proxy-completed DMD-QoL is easy to use, understandable, and face valid for use by caregivers of children aged 4–7 years with DMD. A second phase of interviews should be conducted to further evaluate the measure’s generalizability and accessibility in this age group, including among children aged 4 years with DMD.
期刊介绍:
This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies).
The Editors welcome original articles from all areas of the field:
• Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery).
• Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics.
• Studies of animal models relevant to the human diseases.
The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.