Madeline Schopp , Kelly Beer , Ian Cooper , Kathryn Hird , Althea Doverty , Annik Panicker , Katie Schütze , Anna Brusch , Merrilee Needham
{"title":"包涵体肌炎患者报告和临床评估结果之间的关系——来自回顾性队列研究的见解。","authors":"Madeline Schopp , Kelly Beer , Ian Cooper , Kathryn Hird , Althea Doverty , Annik Panicker , Katie Schütze , Anna Brusch , Merrilee Needham","doi":"10.1016/j.nmd.2024.105272","DOIUrl":null,"url":null,"abstract":"<div><div>Inclusion body myositis (IBM) is an inflammatory myopathy, characterised by slow progression of weakness, skeletal muscle atrophy, and heterogeneous clinical presentation. This variability in disease progression and presentation complicates tracking of clinical progress and intervention response in clinical trials, presenting challenges in identifying reliable outcome measures. We aimed to identify the most useful suite of clinician-assessed and patient-reported outcome measures (PROMs) for use in clinical practice and trials from a selection of the most commonly used outcome measures in IBM. We retrospectively analysed clinician-assessed outcome measures (manual muscle testing (MMT8, MMT12)), right- and left-handed grip strength, modified timed up and go (mTUG), two-minute walk test (2MWT); a clinician-administered patient-reported tool (IBM Functional Rating Scale (IBMFRS)); and PROMs including the eating assessment tool (EAT-10), and neuromuscular symptom score (NSS) from 20 participants attending a single specialist myositis clinic in Perth, Australia. Correlation analysis revealed significant correlations between the IBMFRS, MMT8, MMT12, mTUG and 2MWT (<em>p</em> < 0.05). The NSS strongly correlated with the MMT8, MMT12 and 2MWT (<em>p</em> < 0.05). Univariate regression analyses revealed that 2MWT, MMT12 and mTUG were significant predictors of the IBMFRS and NSS, and backward stepwise linear regression highlighted that the 2MWT was a significant positive predictor for the IBMFRS (<em>p</em> < 0.001). Overall, we concluded that the IBMFRS, NSS, 2MWT and mTUG models were the best predictors of patient-perceived physical function in IBM.</div></div>","PeriodicalId":19135,"journal":{"name":"Neuromuscular Disorders","volume":"46 ","pages":"Article 105272"},"PeriodicalIF":2.7000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The relationship between patient-reported and clinician-assessed outcome measures in Inclusion body myositis - insights from a retrospective cohort study\",\"authors\":\"Madeline Schopp , Kelly Beer , Ian Cooper , Kathryn Hird , Althea Doverty , Annik Panicker , Katie Schütze , Anna Brusch , Merrilee Needham\",\"doi\":\"10.1016/j.nmd.2024.105272\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Inclusion body myositis (IBM) is an inflammatory myopathy, characterised by slow progression of weakness, skeletal muscle atrophy, and heterogeneous clinical presentation. This variability in disease progression and presentation complicates tracking of clinical progress and intervention response in clinical trials, presenting challenges in identifying reliable outcome measures. We aimed to identify the most useful suite of clinician-assessed and patient-reported outcome measures (PROMs) for use in clinical practice and trials from a selection of the most commonly used outcome measures in IBM. We retrospectively analysed clinician-assessed outcome measures (manual muscle testing (MMT8, MMT12)), right- and left-handed grip strength, modified timed up and go (mTUG), two-minute walk test (2MWT); a clinician-administered patient-reported tool (IBM Functional Rating Scale (IBMFRS)); and PROMs including the eating assessment tool (EAT-10), and neuromuscular symptom score (NSS) from 20 participants attending a single specialist myositis clinic in Perth, Australia. Correlation analysis revealed significant correlations between the IBMFRS, MMT8, MMT12, mTUG and 2MWT (<em>p</em> < 0.05). The NSS strongly correlated with the MMT8, MMT12 and 2MWT (<em>p</em> < 0.05). Univariate regression analyses revealed that 2MWT, MMT12 and mTUG were significant predictors of the IBMFRS and NSS, and backward stepwise linear regression highlighted that the 2MWT was a significant positive predictor for the IBMFRS (<em>p</em> < 0.001). Overall, we concluded that the IBMFRS, NSS, 2MWT and mTUG models were the best predictors of patient-perceived physical function in IBM.</div></div>\",\"PeriodicalId\":19135,\"journal\":{\"name\":\"Neuromuscular Disorders\",\"volume\":\"46 \",\"pages\":\"Article 105272\"},\"PeriodicalIF\":2.7000,\"publicationDate\":\"2025-01-01\",\"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/S0960896624017681\",\"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/S0960896624017681","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
The relationship between patient-reported and clinician-assessed outcome measures in Inclusion body myositis - insights from a retrospective cohort study
Inclusion body myositis (IBM) is an inflammatory myopathy, characterised by slow progression of weakness, skeletal muscle atrophy, and heterogeneous clinical presentation. This variability in disease progression and presentation complicates tracking of clinical progress and intervention response in clinical trials, presenting challenges in identifying reliable outcome measures. We aimed to identify the most useful suite of clinician-assessed and patient-reported outcome measures (PROMs) for use in clinical practice and trials from a selection of the most commonly used outcome measures in IBM. We retrospectively analysed clinician-assessed outcome measures (manual muscle testing (MMT8, MMT12)), right- and left-handed grip strength, modified timed up and go (mTUG), two-minute walk test (2MWT); a clinician-administered patient-reported tool (IBM Functional Rating Scale (IBMFRS)); and PROMs including the eating assessment tool (EAT-10), and neuromuscular symptom score (NSS) from 20 participants attending a single specialist myositis clinic in Perth, Australia. Correlation analysis revealed significant correlations between the IBMFRS, MMT8, MMT12, mTUG and 2MWT (p < 0.05). The NSS strongly correlated with the MMT8, MMT12 and 2MWT (p < 0.05). Univariate regression analyses revealed that 2MWT, MMT12 and mTUG were significant predictors of the IBMFRS and NSS, and backward stepwise linear regression highlighted that the 2MWT was a significant positive predictor for the IBMFRS (p < 0.001). Overall, we concluded that the IBMFRS, NSS, 2MWT and mTUG models were the best predictors of patient-perceived physical function in IBM.
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.