期刊
NEUROLOGY
卷 86, 期 15, 页码 1408-1416出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1212/WNL.0000000000002416
关键词
-
资金
- NIH National Institute of Neurological Disorders and Stroke [R01 NS045686]
- Muscular Dystrophy Association [93615]
- NIH NCATS [UL1 TR000042, UL1 TR000040, UL1TR001070, UL1TR000001, UL1TR000124, UL1 TR000004, UL1 TR001105, UL1 TR000135]
- NIH NCRR
- NCATS [UL1 RR024160, UL1 RR024131]
- NIH NCRR [C06 RR20092, M01-02635, 1 UL1 RR025758]
- NIH NINDS [U10NS077384, R37-AR42703, RO1-AR063182]
- MRC Centre Grant
- UCL Biomedical Research Centre Grant
- NINDS
- MDA
- MRC [MR/K000608/1] Funding Source: UKRI
- Medical Research Council [MR/K000608/1] Funding Source: researchfish
Objective:To determine the short-term and long-term effects of dichlorphenamide (DCP) on attack frequency and quality of life in hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis.Methods:Two multicenter randomized, double-blind, placebo-controlled trials lasted 9 weeks (Class I evidence), followed by a 1-year extension phase in which all participants received DCP. Forty-four HOP and 21 HYP participants participated. The primary outcome variable was the average number of attacks per week over the final 8 weeks of the double-blind phase.Results:The median attack rate was lower in HOP participants on DCP than in participants on placebo (0.3 vs 2.4, p = 0.02). The 9-week mean change in the Physical Component Summary score of the Short Form-36 was also better in HOP participants receiving DCP (treatment effect = 7.29 points, 95% confidence interval 2.26 to 12.32, p = 0.006). The median attack rate was also lower in HYP participants on DCP (0.9 vs 4.8) than in participants on placebo, but the difference in median attack rate was not significant (p = 0.10). There were no significant effects of DCP on muscle strength or muscle mass in either trial. The most common adverse events in both trials were paresthesia (47% DCP vs 14% placebo, both trials combined) and confusion (19% DCP vs 7% placebo, both trials combined).Conclusions:DCP is effective in reducing the attack frequency, is safe, and improves quality of life in HOP periodic paralysis.Classification of evidence:These studies provide Class I evidence that DCP significantly reduces attack frequency in HOP but lacked the precision to support either efficacy or lack of efficacy of DCP in HYP.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据