{
  "abstract": "Introduction Patient-reported outcome measures (PROMs) are gaining recognition as potential biomarkers for predicting disease progression and treatment response in clinical trials. The MS-QoL-54, a 54-item questionnaire, was used in the phase 3 SPI-2 study, which evaluated high-dose biotin’s impact on the Extended Disability Status Scale (EDSS) in progressive multiple sclerosis (PMS).This study aimed to determine whether changes in MS-QoL-54 summary scores could predict future EDSS changes and if simulated treatment effects on MS-QoL-54 could reliably support clinical trials.Methods Data from baseline to month 30 (n = 642) were analyzed. Correlation and linear mixed-effects analysis assessed if 15-month PROM change predicted 30-month EDSS changes. Trial simulations evaluated treatment effects up to 50% on sample sizes up to n=500.Results The MS-QoL-54 Physical Score showed the strongest correlation (Spearman’s rho: 0.14, p = 0.03) and predicted EDSS changes via linear mixed-effects model (β = 0.0053, p = 0.002). However, trial simulations demonstrated limited power (max 0.57).Conclusion Although the MS-QoL-54 can predict EDSS changes, its variability limits utility as a reliable trial marker. Future research should prioritize more MS-specific questionnaires, such as the MSIS-29 and MSWS-12, which may better capture meaningful changes in PMS progression and treatment effects.Sean.apapmangion@nhs.net",
  "authors": [
    {
      "affiliations": [
        "UCL"
      ],
      "name": "Apap Mangion Sean"
    },
    {
      "affiliations": [
        "UCL"
      ],
      "name": "Eshaghi Arman"
    },
    {
      "affiliations": [
        "UCL"
      ],
      "name": "Jeremy Chataway"
    }
  ],
  "title": "140 Predicting future disability progression in PMS using the MS-QoL-54",
  "uid": "a1401140-55d0-5637-8f22-e4b794a7c5df"
}