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Ephedrine treatment for autoimmune myasthenia gravis

Lipka, AF; Vrinten, C; van Zwet, EW; Schimmel, KJM; Cornel, MC; Kuijpers, MR; Hekster, YA; ... Verschuuren, JJGM; + view all (2017) Ephedrine treatment for autoimmune myasthenia gravis. Neuromuscular Disorders , 27 (3) pp. 259-265. 10.1016/j.nmd.2016.11.009. Green open access

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Abstract

We studied the effect and safety of ephedrine as add-on treatment for patients with myasthenia gravis with acetylcholine receptor antibodies (AChR MG), who do not sufficiently respond to standard treatment. Four patients with AChR MG were included in a placebo-controlled, double-blind, randomised, multiple crossover series of n-of-1 trials. Each n-of-1 trial consisted of 3 cycles, in which two 5-day intervention periods were followed by 2 days washout. In each cycle, ephedrine 50mg daily in 2 doses was compared with placebo in the alternate treatment period. Primary outcome was a change in QMG score. Add-on treatment with ephedrine compared with placebo improved QMG score by 1.0 point (95% confidence interval 0.21-1.79), which was significant for the group of trial patients as well as for the population treatment effect. Ephedrine also showed a significant trial average treatment effect for all secondary outcomes, improving MG-Composite by 2.7, MG-ADL by 1.0 and VAS score for muscle strength by 1.1. Adverse events were mild and included palpitations, tremor and restlessness. Although all ECGs were normal, ephedrine prolonged the corrected QT interval. Ephedrine as add-on treatment for myasthenia gravis resulted in a small but consistent reduction of symptoms and weakness, in patients with moderate disease severity.

Type: Article
Title: Ephedrine treatment for autoimmune myasthenia gravis
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.nmd.2016.11.009
Publisher version: http://dx.doi.org/10.1016/j.nmd.2016.11.009
Language: English
Additional information: Copyright © 2016 Elsevier B.V. All rights reserved. This manuscript version is made available under the CC-BY-NC-ND 4.0 license http://creativecommons.org/licenses/by-nc-nd/4.0/
Keywords: Myasthenia gravis; add-on therapy; ephedrine; n-of-1 trial; randomised controlled trial
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > Institute of Epidemiology and Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > Institute of Epidemiology and Health > Epidemiology and Public Health
URI: http://discovery.ucl.ac.uk/id/eprint/1529340
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