217P Comparing IBMFRS and sIFA as progression indicators in inclusion body myositis patients from the INSPIRE-IBM trial

IF 2.7 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2024-10-01 DOI:10.1016/j.nmd.2024.07.068

P. Gaid , M. Wencel , I. Hernandez , N. Goyal , M. Dimachkie , T. Lloyd , P. Mohassel , C. Weihl , M. Freimer , A. Shaibani , M. Wicklund , S. Dixon , N. Chahin , L. Wang , P. Shieh , A. Amato , C. Quinn , O. Carbunar , T. Mozaffar , INSPIRE IBM Study Group

{"title":"217P Comparing IBMFRS and sIFA as progression indicators in inclusion body myositis patients from the INSPIRE-IBM trial","authors":"P. Gaid , M. Wencel , I. Hernandez , N. Goyal , M. Dimachkie , T. Lloyd , P. Mohassel , C. Weihl , M. Freimer , A. Shaibani , M. Wicklund , S. Dixon , N. Chahin , L. Wang , P. Shieh , A. Amato , C. Quinn , O. Carbunar , T. Mozaffar , INSPIRE IBM Study Group","doi":"10.1016/j.nmd.2024.07.068","DOIUrl":null,"url":null,"abstract":"<div><div>Inclusion body myositis (IBM) is a common muscular disorder in individuals over the age of 40 years, characterized by atrophy and progressive muscle weakness. Patient-reported outcomes such as the IBMFRS or the sIFA questionnaire provide valuable insights into disease impact from the patient's perspective on their symptoms, functional limitations, and quality of life. However, it remains a topic of further investigation to determine which of these questionnaires exhibits stronger correlations with disease progression. The INSPIRE-IBM is a natural history study involving 150 IBM patients across 13 different US sites. Evaluations are conducted biannually over two years and patients complete IBMFRS, sIFA, EAT-10, Sydney Swallow Questionnaire, PROMIS, along with manual muscle testing and pulmonary functions tests. This abstract analyzes correlations between IBMFRS and sIFA with the other assessments by regression analysis to identify which is a stronger correlator with disease progression. Preliminary analysis, involving 87 patients who completed three time points, revealed a strong correlation between IBMFRS and sIFA (R2=0.7, p=3.21E-96). Both outcomes show moderate correlation with PFTs (R2 between 0.5-0.7), with no significant difference in strength of correlation. IBMFRS and sIFA exhibit similar correlation with MMTs (R2=0.43, p=0.93). As the study is ongoing, more timepoints will be available per patient closer to the conference date and will be included in the analysis.</div></div>","PeriodicalId":19135,"journal":{"name":"Neuromuscular Disorders","volume":"43 ","pages":"Article 104441.59"},"PeriodicalIF":2.7000,"publicationDate":"2024-10-01","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/S0960896624002323","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Inclusion body myositis (IBM) is a common muscular disorder in individuals over the age of 40 years, characterized by atrophy and progressive muscle weakness. Patient-reported outcomes such as the IBMFRS or the sIFA questionnaire provide valuable insights into disease impact from the patient's perspective on their symptoms, functional limitations, and quality of life. However, it remains a topic of further investigation to determine which of these questionnaires exhibits stronger correlations with disease progression. The INSPIRE-IBM is a natural history study involving 150 IBM patients across 13 different US sites. Evaluations are conducted biannually over two years and patients complete IBMFRS, sIFA, EAT-10, Sydney Swallow Questionnaire, PROMIS, along with manual muscle testing and pulmonary functions tests. This abstract analyzes correlations between IBMFRS and sIFA with the other assessments by regression analysis to identify which is a stronger correlator with disease progression. Preliminary analysis, involving 87 patients who completed three time points, revealed a strong correlation between IBMFRS and sIFA (R2=0.7, p=3.21E-96). Both outcomes show moderate correlation with PFTs (R2 between 0.5-0.7), with no significant difference in strength of correlation. IBMFRS and sIFA exhibit similar correlation with MMTs (R2=0.43, p=0.93). As the study is ongoing, more timepoints will be available per patient closer to the conference date and will be included in the analysis.

查看原文本刊更多论文

217P 将 IBMFRS 和 sIFA 作为 INSPIRE-IBM 试验中包涵体肌炎患者的病情进展指标进行比较

包涵体肌炎（IBM）是一种常见的肌肉疾病，多发于 40 岁以上的人群，以肌肉萎缩和进行性肌无力为特征。患者报告结果（如 IBMFRS 或 sIFA 问卷）从患者的角度提供了有关其症状、功能限制和生活质量对疾病影响的宝贵见解。然而，确定这些问卷中哪一个与疾病进展的相关性更强仍是一个需要进一步研究的课题。INSPIRE-IBM 是一项自然史研究，涉及 150 名 IBM 患者，分布在美国 13 个不同的研究机构。每两年进行一次评估，患者需要完成 IBMFRS、sIFA、EAT-10、悉尼吞咽问卷、PROMIS 以及手动肌肉测试和肺功能测试。本摘要通过回归分析，分析了 IBMFRS 和 sIFA 与其他评估之间的相关性，以确定哪项评估与疾病进展的相关性更强。初步分析显示，IBMFRS 和 sIFA 之间有很强的相关性（R2=0.7，p=3.21E-96）。这两项结果均与 PFTs 呈中度相关（R2 在 0.5-0.7 之间），相关强度无显著差异。IBMFRS 和 sIFA 与 MMT 的相关性相似（R2=0.43，P=0.93）。由于研究仍在进行中，在临近会议召开日期时，每位患者将获得更多的时间点，并将纳入分析中。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

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.