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Purpose <p>To estimate the minimal clinically important difference (MCID) for the MG-QOL15R.</p> Methods <p>Data from two multi-country myasthenia gravis studies were used: the ADAPT RCT (n = 157) and the MyRealWorld-MG (MRW-MG) survey (n = 92). MCIDs were estimated using four anchor-based methods: change difference (CD), receiver operating characteristic (ROC) curve with Area Under the Curve (AUC), linear regression, and equipercentile linking. MG-Activities of Daily Living (MG-ADL) was the anchor. MCIDs were applied to the ADAPT RCT to assess the impact of different thresholds. Odds ratios (ORs) and 95% confidence intervals (CIs) were calculated to estimate the odds of achieving an MCID threshold at week 4 in the efgartigimod arm compared with placebo among patients positive for acetylcholine receptor antibodies.</p> Results <p>Baseline mean age was 46.7&#xa0;years old in ADAPT and 49.8&#xa0;years old in MRW-MG; women comprised 71.3% and 71.7%, respectively. ADAPT included only generalized MG, while MRW-MG included 12.0% ocular and 88.0% generalized MG. Average disease severity (MG-ADL 9.0 vs. 6.3) and HRQoL impairment (MG-QOL15R 16.3 vs. 12.0) were greater in ADAPT. MCID estimates were: CD (ADAPT 1.6; MRW-MG 2.8), ROC (both 2.5, AUCs 0.66; 0.71), linear regression (2.1; 1.6), and equipercentile linking (2.0; 3.0). Applying MCIDs of  ≥ 2.0 and ≥ 3.0 resulted in ORs of 3.31 (95%CI 1.65–6.87) and 4.14 (95%CI 2.11–8.38).</p> Conclusions <p>MCID estimates ranged between 1.6 and 3.0 across both studies. MCID thresholds of 2- and 3-points may indicate a minimal clinically meaningful change for monitoring progress and guiding treatment. Future research should use patient global impression of change anchors to improve interpretability and clinical relevance of MCID estimates.</p>

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Estimating the minimal clinically important difference for the Myasthenia Gravis Quality of Life revised scale (MG-QOL15R)

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月13日

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  • Ângela Jornada Ben,
  • Febe Brackx,
  • Glenn Phillips,
  • Carolina Barnett-Tapia,
  • Cynthia Qi,
  • Fanni Rencz,
  • Sarah Dewilde

摘要

Purpose

To estimate the minimal clinically important difference (MCID) for the MG-QOL15R.

Methods

Data from two multi-country myasthenia gravis studies were used: the ADAPT RCT (n = 157) and the MyRealWorld-MG (MRW-MG) survey (n = 92). MCIDs were estimated using four anchor-based methods: change difference (CD), receiver operating characteristic (ROC) curve with Area Under the Curve (AUC), linear regression, and equipercentile linking. MG-Activities of Daily Living (MG-ADL) was the anchor. MCIDs were applied to the ADAPT RCT to assess the impact of different thresholds. Odds ratios (ORs) and 95% confidence intervals (CIs) were calculated to estimate the odds of achieving an MCID threshold at week 4 in the efgartigimod arm compared with placebo among patients positive for acetylcholine receptor antibodies.

Results

Baseline mean age was 46.7 years old in ADAPT and 49.8 years old in MRW-MG; women comprised 71.3% and 71.7%, respectively. ADAPT included only generalized MG, while MRW-MG included 12.0% ocular and 88.0% generalized MG. Average disease severity (MG-ADL 9.0 vs. 6.3) and HRQoL impairment (MG-QOL15R 16.3 vs. 12.0) were greater in ADAPT. MCID estimates were: CD (ADAPT 1.6; MRW-MG 2.8), ROC (both 2.5, AUCs 0.66; 0.71), linear regression (2.1; 1.6), and equipercentile linking (2.0; 3.0). Applying MCIDs of  ≥ 2.0 and ≥ 3.0 resulted in ORs of 3.31 (95%CI 1.65–6.87) and 4.14 (95%CI 2.11–8.38).

Conclusions

MCID estimates ranged between 1.6 and 3.0 across both studies. MCID thresholds of 2- and 3-points may indicate a minimal clinically meaningful change for monitoring progress and guiding treatment. Future research should use patient global impression of change anchors to improve interpretability and clinical relevance of MCID estimates.