TY - JOUR
T1 - Ephedrine treatment for autoimmune myasthenia gravis
AU - Lipka, Alexander F.
AU - Vrinten, Charlotte
AU - van Zwet, Erik W.
AU - Schimmel, Kirsten J.M.
AU - Cornel, Martina C.
AU - Kuijpers, Marja R.
AU - Hekster, Yechiel A.
AU - Weinreich, Stephanie S.
AU - Verschuuren, Jan J.G.M.
PY - 2017/3/1
Y1 - 2017/3/1
N2 - 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, and 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 50 mg 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.
AB - 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, and 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 50 mg 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.
KW - Add-on therapy
KW - Ephedrine
KW - Myasthenia gravis
KW - Randomised controlled trial
KW - n-of-1 trial
UR - http://www.scopus.com/inward/record.url?scp=85008392783&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.nmd.2016.11.009
DO - https://doi.org/10.1016/j.nmd.2016.11.009
M3 - Article
C2 - 28007405
SN - 0960-8966
VL - 27
SP - 259
EP - 265
JO - Neuromuscular disorders
JF - Neuromuscular disorders
IS - 3
ER -