Abstract
An increasing number of clinical trials are enrolling patients with
myasthenia gravis (MG). A lack of standardization in the performance of
outcome measures leads to confusion among site research teams and is a
source of variability in clinical trial data. MGNet, the NIH-supported
Rare Disease Clinical Research Network for MG, views standardization of
MG outcome measures as a critical need. To address this issue, a group
of experts summarized key outcome measures used in MG clinical trials
and a symposium was convened to address issues contributing to outcome
measure variability. Consensus recommendations resulted in changes to
outcome measure instructions and, in some cases, modifications to
specific instruments. Recommended changes were posted for public
commentary before finalization. Changes to the MG-Activities of Daily
Living, MG-Quality of Life-15r, and MG-Impairment Index were limited to
adding details to the administration instructions. Recommendations for
proper positioning of participants and how to score items that could not
be performed because of non-MG reasons were provided for the MG
Composite. The Quantitative MG (QMG) score required the most attention,
and changes were made both to the instructions and the performance of
certain items resulting in the QMG-Revised. The Postintervention Status
was believed to have a limited role in clinical trials, except for the
concept of minimal manifestation status. As a next step, training
materials and revised source documents, which will be freely available
to study teams, will be created and posted on the MGNet website. Further
studies are needed to validate changes made to the QMG-Revised.
