Trial reportNeurology2016
Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis.
Open access · bronzeAbstract readMulticenter StudyRandomized Controlled Trial
In one paragraph
Trial report in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 2 pooled it
5.0field-weighted citation impact, top 4% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
31 citing papers in PubMed, 2 syntheses or guidelines pooled it, 70 citations in OpenAlex.
- Targeted Therapies for Skeletal Muscle Ion Channelopathies: Systematic Review and Steps Towards Precision Medicine.Journal of neuromuscular diseases · 2021Pooled it
- Side effects of acetazolamide: a systematic review and meta-analysis assessing overall risk and dose dependence.BMJ open respiratory research · 2020Pooled it
- Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis.Muscle & nerve · 2021Trial
- Open-Label, Dose-Escalation, Phase 1 Study of Safety and Single and Multiple-Dose Pharmacokinetics of Dichlorphenamide in Healthy Volunteers.Clinical pharmacology in drug development · 2019Trial
- Review
- Article
- Permanent weakness and myopathy in hypokalemic periodic paralysis.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2026Review
- Lifestyle and dietary measures in Periodic Paralyses.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2026Review
- Hypokalemic Periodic Paralysis Associated With a RareCase reports in genetics · 2026Article
- Molecular genetics of skeletal muscle channelopathies.Journal of human genetics · 2025Review
- Quantitative Muscle MRI to Monitor Disease Progression in Hypokalemic Period Paralysis.Neurology. Genetics · 2024Article
- Functional effects of drugs and toxins interacting with NaFrontiers in pharmacology · 2024Review
- Hypokalemic periodic paralysis: a 3-year follow-up study.Journal of neurology · 2023Article
- Muscle channelopathies: A review.Annals of the Child Neurology Society · 2023Review
- Optical measurement of gating pore currents in hypokalemic periodic paralysis model cells.Disease models & mechanisms · 2023Article
- Retigabine suppresses loss of force in mouse models of hypokalaemic periodic paralysis.Brain : a journal of neurology · 2023Article
- The role of nephrologists in management of hypokalemic periodic paralysis: a case report.Journal of medical case reports · 2022Article
- Case report: SCN4A p.R1135H gene variant in combination with thyrotoxicosis causing hypokalemic periodic paralysis.Frontiers in neurology · 2022Article
- Ion Channel Gene Mutations Causing Skeletal Muscle Disorders: Pathomechanisms and Opportunities for Therapy.Cells · 2021Review
- The Role of Nutrition and Physical Activity as Trigger Factors of Paralytic Attacks in Primary Periodic Paralysis.Journal of neuromuscular diseases · 2021Review
Corrections and comments
- Commented on by
Authors and funding
23 authors at 11 institutions in 2 countries.
Valeria A SansoneFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD. valeria.sansone@unimi.it.
James BurgeFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Michael P McDermottFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Patty C SmithFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Barbara HerrFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Rabi TawilFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Shree PandyaFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
John KisselFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Emma CiafaloniFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Perry ShiehFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Jeffrey W RalphFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Antony AmatoFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Steve C CannonFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Jaya TrivediFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Richard BarohnFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Brian CrumFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Hiroshi MitsumotoFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Alan PestronkFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Giovanni MeolaFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Robin ConwitFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Michael G HannaFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Robert C GriggsFrom NEMO Clinical Center (V.A.S.) and IRCCS Policlinico San Donato (G.M.), University of Milan, Italy; MRC Centre for Neuromuscular Diseases (J.B., M.G.H.), UCL Institute of Neurology, Queen Square, London, UK; University of Rochester (M.P.M., P.C.S., B.H., R.T., S.P., E.C., R.C.G.), NY; Ohio State University (J.K.), Columbus; UCLA Medical Center (P.S.), Los Angeles, CA; University of California San Francisco School of Medicine (J.W.R.); Brigham and Women's Hospital (A.A.), Boston, MA; UT Southwestern Medical Center (S.C.C., J.T.), Dallas, TX; University of Kansas Medical Center (R.B.), Kansas City; Mayo Clinic (B.C.), Rochester MN; Columbia University (H.M.), New York, NY; Washington University (A.P.), St. Louis, MO; and the Office of Clinical Research (R.C.), NINDS, Bethesda, MD.
Muscle Study Group
IRCCS Policlinico San Donato · ITMayo Clinic in Arizona · USSouthwestern Medical CenterUniversity of Rochester · USBrigham and Women's Hospital · USThe Ohio State University · USThe University of Texas Southwestern Medical Center · USUCLA Medical Center · USUniversity of California, San Francisco · USUniversity of Kansas Medical Center · USWashington University in St. Louis · US
Funding
Disease Pathogenesis and Modification for CaV1.1-Associated Hypokalemic Periodic ParalysisR01AR063182 · NIAMS · UT SOUTHWESTERN MEDICAL CENTER · PI STEPHEN C. CANNON · 2012 to 2026
$6.0MMolecular Physiology of Myotonia and Periodic ParalysisR37AR042703 · NIAMS · UT SOUTHWESTERN MEDICAL CENTER · PI CANNON, STEPHEN C. · 2008 to 2018
$4.2MMedical Research Council MR/K000608/1NIAMS NIH HHS R01 AR063182NIAMS NIH HHS R37 AR042703
Abstract
objectiveTo 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.
methodsTwo 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.
resultsThe 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).
conclusionsDCP 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.
Indexed as
AdultCarbonic Anhydrase InhibitorsDichlorphenamideDouble-Blind MethodFemaleFollow-Up StudiesHumansMaleMiddle AgedParalyses, Familial PeriodicCarbonic Anhydrase InhibitorsDichlorphenamide
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