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. 2020 Sep;22(1):11-21.
doi: 10.1097/CND.0000000000000301.

Longitudinal Analysis of Disease Burden in Refractory and Nonrefractory Generalized Myasthenia Gravis in the United States

Linda Harris  1 , Phillip H Allman  2 , Reinee Sheffield  3 , Gary Cutter  2
Affiliations

Longitudinal Analysis of Disease Burden in Refractory and Nonrefractory Generalized Myasthenia Gravis in the United States

Linda Harris et al. J Clin Neuromuscul Dis. 2020 Sep.

Abstract

Objective: To compare temporal trends in clinical and health care resource utilization (HRU) outcomes in people with refractory and nonrefractory generalized myasthenia gravis (gMG).

Methods: A retrospective analysis of data from adults with gMG in the Myasthenia Gravis Foundation of America Patient Registry. gMG status (ever-refractory or always nonrefractory) and clinical (Myasthenia Gravis-Activities of Daily Living [MG-ADL] scores, exacerbations) and HRU outcomes were determined from questionnaires self-completed 6-monthly for up to 4 years. The probability of each outcome was compared for the 2 groups over time.

Results: The mean MG-ADL score and the probability of experiencing each outcome were significantly greater in the ever-refractory versus nonrefractory groups during each year of follow-up. Between-group differences in time trends were statistically significant for intensive care and feeding-tube use.

Conclusions: People who have ever had refractory gMG may have worse functional status, more exacerbations, and higher HRU than people with consistently nonrefractory disease.

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Figures

FIGURE 1.
FIGURE 1.
Predicted results from generalized estimating equation models according to refractory status* over time for: (A) MG-ADL total scores†; (B) probability of any exacerbation‡; (C) number of exacerbations‡; (D) probability of any ED visit‡; (E) number of ED visits‡; (F) probability of any overnight hospitalization‡; (G) probability of any ICU use‡,§; and (H) probability of any feeding-tube use. ‡,§ Sample means are displayed as dots. Shading indicates 95% CI. *Participants were included in the ever-refractory group from the point at which their disease was defined as refractory; participants were included in the nonrefractory group if their disease was nonrefractory at enrollment and at all timepoints assessed during the study. †Over the previous 4 weeks. ‡In the previous 6 months. §Results should be interpreted with caution owing to the small number of participants who provided data between years 3 and 4.
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