期刊
MUSCLE & NERVE
卷 62, 期 2, 页码 261-265出版社
WILEY
DOI: 10.1002/mus.26910
关键词
MG-ADL; MGTX; myasthenia gravis; outcome measure; QMG; trial design
资金
- Myasthenia Gravis Foundation of America
- National Institute of Neurological Disorders and Stroke [U01 NS042685]
- Ra Pharmaceuticals, Inc.
- American Academy of Neurology
- Alexion Pharmaceuticals
- Muscular Dystrophy Association
Introduction Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scales were compared using the data from the Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy (MGTX) study. Methods Correlation between QMG and MG-ADL raw and change-from-baseline scores was calculated every 3 months for 60 months based on treatment groups and minimal manifestation status (MMS). Results QMG and MG-ADL change-from-baseline scores correlated significantly, with increasing strength of correlation over time, in both treatment groups. QMG and MG-ADL raw scores correlated significantly in both treatment groups, with increasing correlation only in the prednisone-alone group. Correlation between raw scores was weaker in patients who were in MMS, demonstrating a floor effect on the MG-ADL scale. Raw QMG scores could be modeled assuming a normal distribution, whereas raw MG-ADL scores could not be modeled this way. Discussion The floor effect and skewed distribution of the MG-ADL measure should be taken into account in the design of myasthenia gravis clinical trials.
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