RT Journal Article
SR Electronic
T1 Long-term Safety and Efficacy of Mexiletine in Myotonic Dystrophy Types 1 and 2
JF Neurology: Clinical Practice
FD Lippincott Williams & Wilkins
SP e682
OP e685
DO 10.1212/CPJ.0000000000001073
VO 11
IS 5
A1 Mousele, Christina
A1 Matthews, Emma
A1 Pitceathly, Robert D.S.
A1 Hanna, Michael G.
A1 MacDonald, Susan
A1 Savvatis, Konstantinos
A1 Carr, Aisling
A1 Turner, Christopher
YR 2021
UL http://cp.neurology.org/content/11/5/e682.abstract
AB Background and Objective Myotonic dystrophy types 1 and 2 are progressive multisystem genetic disorders whose core clinical feature is myotonia. Mexiletine, an antagonist of voltage-gated sodium channels, is a recommended antimyotonic agent in the nondystrophic myotonias, but its use in myotonic dystrophy is limited because of lack of data regarding its long-term efficacy and safety profile.Methods To address this issue, this study retrospectively evaluated patients with myotonic dystrophy receiving mexiletine over a mean time period of 32.9 months (range 0.1–216 months).Results This study demonstrated that 96% of patients reported some improvement in myotonia symptoms with mexiletine treatment. No clinically relevant cardiac adverse events were associated with the long-term use of mexiletine.Conclusions These findings support that mexiletine is both safe and effective when used long-term in myotonic dystrophy.Classification of Evidence This study provides Class IV evidence that mexiletine is a well-tolerated and effective treatment for myotonic dystrophy types 1 and 2.