{"title":"测量CIDP治疗效果的最小临床重要差异:历史,当前使用,局限性和前景。","authors":"Yusuf A Rajabally, Kabir K Nazeer, Young Gi Min","doi":"10.1002/mus.28478","DOIUrl":null,"url":null,"abstract":"<p><p>Outcome measures are essential for evaluating treatment effects and disease progression in chronic inflammatory demyelinating polyneuropathy (CIDP). The concept of the minimal clinically important difference (MCID), which represents the smallest change in a measure deemed clinically meaningful, has become increasingly important in CIDP research, and is also gaining interest in clinical practice. This review explores the history of use in clinical trials and observational studies, as well as potential limitations and future perspectives for MCIDs in CIDP. MCID derivation methods include anchor-based approaches that rely on patient perspectives, and distribution-based methods that calculate the magnitude of changes exceeding statistical error margins. Both approaches have been used in CIDP, yielding MCID cut-offs for key scales such as the Inflammatory Neuropathy Cause and Treatment (INCAT) Scale, Overall Neuropathy Limitation Scale (ONLS), Rasch-built Overall Disability Scale (I-RODS), grip strength, and the Medical Research Council sum score. Challenges include discrepancies in MCID thresholds, particularly for I-RODS and strength measures, and variability related to disease severity and subtype. Despite these issues, MCIDs for disability measures such as INCAT, ONLS, and I-RODS have demonstrated their value through validity and clinical relevance, making them suitable for both research and clinical practice. MCIDs for strength scores, walking tests, sensory scales, and electrophysiological measures lack reliability and direct clinical relevance with regard to the primary concept of clinically meaningful benefit. Future research should focus on optimization of outcome measures, harmonization of MCID derivation methods, and exploration of MCID application with disease-specific Health Related-Quality of Life measures for CIDP.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"1042-1051"},"PeriodicalIF":3.1000,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12529031/pdf/","citationCount":"0","resultStr":"{\"title\":\"Minimal Clinically Important Differences in Measuring Treatment Effects in CIDP: History, Current Use, Limitations, and Prospects.\",\"authors\":\"Yusuf A Rajabally, Kabir K Nazeer, Young Gi Min\",\"doi\":\"10.1002/mus.28478\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Outcome measures are essential for evaluating treatment effects and disease progression in chronic inflammatory demyelinating polyneuropathy (CIDP). The concept of the minimal clinically important difference (MCID), which represents the smallest change in a measure deemed clinically meaningful, has become increasingly important in CIDP research, and is also gaining interest in clinical practice. This review explores the history of use in clinical trials and observational studies, as well as potential limitations and future perspectives for MCIDs in CIDP. MCID derivation methods include anchor-based approaches that rely on patient perspectives, and distribution-based methods that calculate the magnitude of changes exceeding statistical error margins. Both approaches have been used in CIDP, yielding MCID cut-offs for key scales such as the Inflammatory Neuropathy Cause and Treatment (INCAT) Scale, Overall Neuropathy Limitation Scale (ONLS), Rasch-built Overall Disability Scale (I-RODS), grip strength, and the Medical Research Council sum score. Challenges include discrepancies in MCID thresholds, particularly for I-RODS and strength measures, and variability related to disease severity and subtype. Despite these issues, MCIDs for disability measures such as INCAT, ONLS, and I-RODS have demonstrated their value through validity and clinical relevance, making them suitable for both research and clinical practice. MCIDs for strength scores, walking tests, sensory scales, and electrophysiological measures lack reliability and direct clinical relevance with regard to the primary concept of clinically meaningful benefit. 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引用次数: 0
摘要
评价慢性炎症性脱髓鞘性多神经病变(CIDP)的治疗效果和疾病进展的结果指标是必不可少的。最小临床重要差异(minimum clinical important difference, MCID)的概念,代表了被认为有临床意义的指标的最小变化,在CIDP研究中变得越来越重要,在临床实践中也越来越受到关注。这篇综述探讨了MCIDs在CIDP临床试验和观察性研究中的使用历史,以及潜在的局限性和未来的前景。MCID的推导方法包括基于锚点的方法(依赖于患者的观点)和基于分布的方法(计算超出统计误差范围的变化幅度)。这两种方法都被用于CIDP,得出关键量表如炎症性神经病变病因和治疗(INCAT)量表、整体神经病变限制量表(ONLS)、Rasch-built整体残疾量表(I-RODS)、握力和医学研究委员会总分等的MCID截止值。挑战包括MCID阈值的差异,特别是i - rod和强度测量,以及与疾病严重程度和亚型相关的变异性。尽管存在这些问题,INCAT、ONLS和i - rod等用于残疾测量的mcid已经通过有效性和临床相关性证明了它们的价值,使它们适用于研究和临床实践。力量评分、行走测试、感觉量表和电生理测量的MCIDs缺乏可靠性,在临床意义获益的主要概念方面缺乏直接的临床相关性。未来的研究应侧重于优化结果测量,统一MCID衍生方法,探索将MCID应用于CIDP的疾病特异性健康相关生活质量测量。
Minimal Clinically Important Differences in Measuring Treatment Effects in CIDP: History, Current Use, Limitations, and Prospects.
Outcome measures are essential for evaluating treatment effects and disease progression in chronic inflammatory demyelinating polyneuropathy (CIDP). The concept of the minimal clinically important difference (MCID), which represents the smallest change in a measure deemed clinically meaningful, has become increasingly important in CIDP research, and is also gaining interest in clinical practice. This review explores the history of use in clinical trials and observational studies, as well as potential limitations and future perspectives for MCIDs in CIDP. MCID derivation methods include anchor-based approaches that rely on patient perspectives, and distribution-based methods that calculate the magnitude of changes exceeding statistical error margins. Both approaches have been used in CIDP, yielding MCID cut-offs for key scales such as the Inflammatory Neuropathy Cause and Treatment (INCAT) Scale, Overall Neuropathy Limitation Scale (ONLS), Rasch-built Overall Disability Scale (I-RODS), grip strength, and the Medical Research Council sum score. Challenges include discrepancies in MCID thresholds, particularly for I-RODS and strength measures, and variability related to disease severity and subtype. Despite these issues, MCIDs for disability measures such as INCAT, ONLS, and I-RODS have demonstrated their value through validity and clinical relevance, making them suitable for both research and clinical practice. MCIDs for strength scores, walking tests, sensory scales, and electrophysiological measures lack reliability and direct clinical relevance with regard to the primary concept of clinically meaningful benefit. Future research should focus on optimization of outcome measures, harmonization of MCID derivation methods, and exploration of MCID application with disease-specific Health Related-Quality of Life measures for CIDP.
期刊介绍:
Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.