Dataset Information

Open-label trial of ranolazine for the treatment of myotonia congenita.

ABSTRACT:

Objective

To determine open-label, pilot study whether ranolazine could improve signs and symptoms of myotonia and muscle stiffness in patients with myotonia congenita (MC).

Methods

Thirteen participants were assessed at baseline and 2, 4, and 5 weeks. Ranolazine was started after baseline assessment (500 mg twice daily), increased as tolerated after week 2 (1,000 mg twice daily), and maintained until week 4. Outcomes included change from baseline to week 4 in self-reported severity of symptoms (stiffness, weakness, and pain), Timed Up and Go (TUG), hand grip and eyelid myotonia, and myotonia on EMG.

Results

Self-reported severity of stiffness (p < 0.0001) and weakness (p < 0.01) was significantly improved compared with baseline. TUG and grip myotonia times were reduced (p = 0.03, p = 0.01). EMG of the abductor digiti minimi and tibialis anterior showed significantly reduced myotonia duration (p < 0.001, p < 0.01) at week 4. No participant discontinued ranolazine because of side effects.

Conclusions

Ranolazine appeared to be well tolerated over a period of 4 weeks in individuals with MC, and ranolazine resulted in improvement of signs and symptoms of muscle stiffness. The findings of this study suggest that ranolazine should be investigated in a larger controlled study.

Classification of evidence

This study provides Class IV evidence that ranolazine improves myotonia in myotonia congenita.

SUBMITTER: Arnold WD

PROVIDER: S-EPMC5562961 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:Background: Persistent angina is prevalent in women, who more often present with atypical angina, and experience less relief from antianginal therapies. The impact of ranolazine on female-specific angina is unclear. A single-arm, open-label trial was conducted to quantify the impact of ranolazine on angina in women with ischemic heart disease (IHD). Materials and Methods: Women with IHD and ≥2 angina episodes/week were recruited from 30 U.S. sites. Angina and nitroglycerin (NTG) consumption were assessed using patient-reported diaries, Seattle Angina Questionnaire (SAQ), Duke Activity Score Index (DASI), and Women's Ischemia Symptom Questionnaire (WISQ) at baseline and at 4 weeks of treatment with ranolazine 500 mg twice/day. A modified intent-to-treat analysis and parametric or nonparametric methods were used as appropriate to analyze changes. Results: Of 171 women enrolled, mean age was 65 ± 12 years. Of the 159 women included in the analysis, at week 4 compared to baseline, median angina frequency decreased with ranolazine treatment from 5.0 to 1.5 attacks/week and median change from baseline was -3.3 (95% confidence interval [CI]: -4.0 to -2.5; p ≤ 0.0001). Median NTG consumption decreased from 2.0 to 0.0 per week over the 4 weeks and median change was -1.0 (95% CI: -2.0 to -0.5; p < 0.0001). All five SAQ subscales showed mean improvements: physical limitation 9.2 (standard error [SE] 1.5; p < 0.0001), angina stability 31.8 (SE 2.7; p < 0.0001), angina frequency 17.7 (SE 1.6; p < 0.0001), treatment satisfaction 9.3 (SE 1.6; p < 0.0001), and disease perception 2.9 (SE 0.8; p < 0.0001). DASI score also improved 2.9 (SE 0.8; p = 0.0014). WISQ subscales also showed significant improvements (all p < 0.0001). Thirty-one women reported drug-related adverse events (AEs), predominantly mild to moderate gastrointestinal symptoms. Conclusions: Women with IHD treated with ranolazine for 4 weeks experienced less angina measured by SAQ and WISQ. NTG use decreased, physical activity improved, and treatment satisfaction improved. AEs were consistent with prior reports.