Minimal Clinically Important Differences in Measuring Treatment Effects in CIDP: History, Current Use, Limitations and Prospects

Abstract

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.