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Identifying a patient-centered outcome measure for a comparative effectiveness treatment trial in myasthenia gravis.

Pushpa NarayanaswamiDonald B SandersKathie BibeauAndrew KruegerJurgen VenitzJeffrey T Guptill
Published in: Muscle & nerve (2021)
Based on patient input, the 15-item Myasthenia Gravis Quality of Life-Revised (MG-QOL15R) scale, a validated patient-reported outcome measure (PRO), was selected as the primary PCOM for PROMISE-MG. Avoiding hospitalization and having minimal to no treatment adverse effects were selected as additional outcome measures. The patient-centeredness of a PRO depends on the context of a study: PROs should be evaluated for appropriateness as a PCOM for every study.
Keyphrases
  • myasthenia gravis
  • patient reported outcomes
  • clinical trial
  • combination therapy
  • big data
  • phase iii
  • deep learning
  • psychometric properties
  • double blind