Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis

Valeria A. Sansone, James Burge, Michael P. McDermott, Patty C. Smith, Barbara Herr, Rabi Tawil, Shree Pandya, John Kissel, Emma Ciafaloni, Perry Shieh, Jeffrey W. Ralph, Antony Amato, Steve C. Cannon, Jaya Trivedi, Richard Barohn, Brian Crum, Hiroshi Mitsumoto, Alan Pestronk, Giovanni Meola, Robin ConwitMichael G. Hanna, Robert C. Griggs

Research output: Contribution to journalArticle

29 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1408-1416
Number of pages9
JournalNeurology
Volume86
Issue number15
DOIs
StatePublished - Apr 12 2016

ASJC Scopus subject areas

  • Clinical Neurology

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    Sansone, V. A., Burge, J., McDermott, M. P., Smith, P. C., Herr, B., Tawil, R., Pandya, S., Kissel, J., Ciafaloni, E., Shieh, P., Ralph, J. W., Amato, A., Cannon, S. C., Trivedi, J., Barohn, R., Crum, B., Mitsumoto, H., Pestronk, A., Meola, G., ... Griggs, R. C. (2016). Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis. Neurology, 86(15), 1408-1416. https://doi.org/10.1212/WNL.0000000000002416