Outcome measures in CIDP: A scoping and mapping review

IF 3.2 3区医学 Q1 CLINICAL NEUROLOGY

Journal of the Neurological Sciences Pub Date : 2025-08-12 DOI:10.1016/j.jns.2025.123654

Yusuf A. Rajabally , Giorgio Maria Boggia , Danielle Riley , Sam Riley , Judith Peatman , Wim Noel , Charlotte Gary , Eduardo Nobile-Orazio

{"title":"Outcome measures in CIDP: A scoping and mapping review","authors":"Yusuf A. Rajabally , Giorgio Maria Boggia , Danielle Riley , Sam Riley , Judith Peatman , Wim Noel , Charlotte Gary , Eduardo Nobile-Orazio","doi":"10.1016/j.jns.2025.123654","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>CIDP is a rare autoimmune disorder with varying symptom presentations. This heterogenous nature challenges standardizing disease monitoring and assessing treatment response. This study aimed to provide a comprehensive overview of clinical outcome assessments (COAs) used in CIDP.</div></div><div><h3>Methods</h3><div>A structured literature review and mapping exercise was conducted to identify COAs reported in CIDP literature published between 2010 and 2023. Supplementary searches were conducted in other relevant sources. Key publications pre-2010 were identified through handsearching or consultation with clinical experts. Clinical experts assisted with prioritization of identified COAs to those clinically relevant to CIDP.</div></div><div><h3>Results</h3><div>Overall, 2027 records were included, with 904 prioritized for COA mapping, and 524 reporting on relevant CIDP COAs. Overall, 99 different COAs were captured; the most frequent related to functional impairment, followed by humanistic burden. Three COAs were most prominent: INCAT, MRC score and I-RODS (reported in 45.6 %, 37.4 % and 29.2 % of publications respectively). Numerous CIDP biomarkers were identified; 21 publications reported on neurofilament light chain (NfL).</div></div><div><h3>Conclusion</h3><div>Substantial heterogeneity in CIDP COAs use exists. This review identified the INCAT, I-RODS, and MRC score as core measures that evaluate important, although not exhaustive, aspects of CIDP disease experience. The absence of validated CIDP biomarkers, and the heterogeneity of available COAs, may impact the assessment of CIDP treatment effectiveness. More research is needed to establish a set of comprehensive outcome measures, that are relevant to clinicians and patients with CIDP.</div></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":"477 ","pages":"Article 123654"},"PeriodicalIF":3.2000,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of the Neurological Sciences","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0022510X25002746","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Background

CIDP is a rare autoimmune disorder with varying symptom presentations. This heterogenous nature challenges standardizing disease monitoring and assessing treatment response. This study aimed to provide a comprehensive overview of clinical outcome assessments (COAs) used in CIDP.

Methods

A structured literature review and mapping exercise was conducted to identify COAs reported in CIDP literature published between 2010 and 2023. Supplementary searches were conducted in other relevant sources. Key publications pre-2010 were identified through handsearching or consultation with clinical experts. Clinical experts assisted with prioritization of identified COAs to those clinically relevant to CIDP.

Results

Overall, 2027 records were included, with 904 prioritized for COA mapping, and 524 reporting on relevant CIDP COAs. Overall, 99 different COAs were captured; the most frequent related to functional impairment, followed by humanistic burden. Three COAs were most prominent: INCAT, MRC score and I-RODS (reported in 45.6 %, 37.4 % and 29.2 % of publications respectively). Numerous CIDP biomarkers were identified; 21 publications reported on neurofilament light chain (NfL).

Conclusion

Substantial heterogeneity in CIDP COAs use exists. This review identified the INCAT, I-RODS, and MRC score as core measures that evaluate important, although not exhaustive, aspects of CIDP disease experience. The absence of validated CIDP biomarkers, and the heterogeneity of available COAs, may impact the assessment of CIDP treatment effectiveness. More research is needed to establish a set of comprehensive outcome measures, that are relevant to clinicians and patients with CIDP.

查看原文本刊更多论文

CIDP的结果测量：范围和绘图审查

背景：cidp是一种罕见的自身免疫性疾病，具有多种症状表现。这种异质性对标准化疾病监测和评估治疗反应提出了挑战。本研究旨在全面概述临床结果评估（COAs）在CIDP中的应用。方法对2010 - 2023年间发表的CIDP文献中的coa进行结构化文献综述和制图。在其他相关来源中进行了补充搜索。通过手工检索或咨询临床专家确定2010年前的主要出版物。临床专家协助确定与CIDP临床相关的coa的优先顺序。结果共纳入2027条记录，其中904条优先用于COA制图，524条报告了相关CIDP COA。总共捕获了99个不同的coa；最常见的与功能障碍有关，其次是人文负担。三个coa最突出：INCAT， MRC评分和I-RODS（分别在45.6%，37.4%和29.2%的出版物中报道）。鉴定了许多CIDP生物标志物；神经丝轻链（NfL）相关文献21篇。结论CIDP COAs的使用存在较大的异质性。本综述将INCAT、I-RODS和MRC评分确定为评估CIDP疾病经历的重要（尽管不是详尽的）方面的核心指标。缺乏经过验证的CIDP生物标志物，以及可用coa的异质性，可能会影响CIDP治疗效果的评估。需要更多的研究来建立一套与临床医生和CIDP患者相关的综合结果测量。

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

Journal of the Neurological Sciences 医学-临床神经学

CiteScore

7.60

自引率

2.30%

发文量

313

审稿时长

22 days

期刊介绍： The Journal of the Neurological Sciences provides a medium for the prompt publication of original articles in neurology and neuroscience from around the world. JNS places special emphasis on articles that: 1) provide guidance to clinicians around the world (Best Practices, Global Neurology); 2) report cutting-edge science related to neurology (Basic and Translational Sciences); 3) educate readers about relevant and practical clinical outcomes in neurology (Outcomes Research); and 4) summarize or editorialize the current state of the literature (Reviews, Commentaries, and Editorials). JNS accepts most types of manuscripts for consideration including original research papers, short communications, reviews, book reviews, letters to the Editor, opinions and editorials. Topics considered will be from neurology-related fields that are of interest to practicing physicians around the world. Examples include neuromuscular diseases, demyelination, atrophies, dementia, neoplasms, infections, epilepsies, disturbances of consciousness, stroke and cerebral circulation, growth and development, plasticity and intermediary metabolism.