Correlation of Quantitative Myasthenia Gravis and Myasthenia Gravis Activities of Daily Living scales in the MGTX study

被引:19
|
作者
McPherson, Tarrant [1 ]
Aban, Inmaculada [1 ]
Duda, Petra W. [2 ]
Farzaneh-Far, Ramin [2 ]
Wolfe, Gil, I [3 ]
Kaminski, Henry J. [4 ]
Cutter, Gary [1 ]
Lee, Ikjae [5 ]
机构
[1] Univ Alabama Birmingham, Dept Biostat, Birmingham, AL 35233 USA
[2] Ra Pharmaceut, Cambridge, MA USA
[3] Univ Buffalo State Univ New York, Jacobs Sch Med & Biomed Sci, Dept Neurol, Buffalo, NY USA
[4] George Washington Univ, Sch Med & Hlth Sci, Dept Neurol, Washington, DC 20052 USA
[5] Univ Alabama Birmingham, Dept Neurol, 200 Sparks Ctr,1720 7th Ave South, Birmingham, AL 35233 USA
关键词
MG-ADL; MGTX; myasthenia gravis; outcome measure; QMG; trial design; MYCOPHENOLATE-MOFETIL; RANDOMIZED-TRIAL; ECULIZUMAB;
D O I
10.1002/mus.26910
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
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.
引用
收藏
页码:261 / 265
页数:5
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