Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis

被引:43
|
作者
Sansone, Valeria A. [1 ]
Burge, James [3 ]
McDermott, Michael P. [4 ]
Smith, Patty C. [4 ]
Herr, Barbara [4 ]
Tawil, Rabi [4 ]
Pandya, Shree [4 ]
Kissel, John [5 ]
Ciafaloni, Emma [4 ]
Shieh, Perry [6 ]
Ralph, Jeffrey W. [7 ]
Amato, Antony [8 ]
Cannon, Steve C. [9 ]
Trivedi, Jaya [9 ]
Barohn, Richard [10 ]
Crum, Brian [11 ]
Mitsumoto, Hiroshi [12 ]
Pestronk, Alan [13 ]
Meola, Giovanni [2 ]
Conwit, Robin [14 ]
Hanna, Michael G. [3 ]
Griggs, Robert C. [4 ]
机构
[1] Univ Milan, NEMO Clin Ctr, I-20122 Milan, Italy
[2] Univ Milan, IRCCS Policlin San Donato, I-20122 Milan, Italy
[3] UCL Inst Neurol, Ctr Neuromuscular Dis, MRC, Queen Sq, London, England
[4] Univ Rochester, Rochester, NY 14627 USA
[5] Ohio State Univ, Columbus, OH 43210 USA
[6] Univ Calif Los Angeles, Med Ctr, Los Angeles, CA 90024 USA
[7] Univ Calif San Francisco, Sch Med, San Francisco, CA 94143 USA
[8] Brigham & Womens Hosp, 75 Francis St, Boston, MA 02115 USA
[9] Univ Texas SW Med Ctr Dallas, Dallas, TX 75390 USA
[10] Univ Kansas, Med Ctr, Lawrence, KS 66045 USA
[11] Mayo Clin, Rochester, MN USA
[12] Columbia Univ, New York, NY USA
[13] Washington Univ, St Louis, MO USA
[14] NINDS, Off Clin Res, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
关键词
SKELETAL-MUSCLE CHANNELOPATHIES; SODIUM-CHANNEL MUTATIONS; HEALTH SURVEY SF-36; ACETAZOLAMIDE TREATMENT; GENE-MUTATIONS; WEAKNESS; INACTIVATION; PHENOTYPE; DYSTROPHY; DIAGNOSIS;
D O I
10.1212/WNL.0000000000002416
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
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.
引用
收藏
页码:1408 / 1416
页数:9
相关论文
共 50 条
  • [41] Sutimlimab in patients with cold agglutinin disease results of the randomized placebo-controlled phase 3 CADENZA trial
    Roeth, Alexander
    Berentsen, Sigbjorn
    Barcellini, Wilma
    D'Sa, Shirley
    Jilma, Bernd
    Michel, Marc
    Weitz, Ilene C.
    Yamaguchi, Masaki
    Nishimura, Jun-ichi
    Vos, Josephine M., I
    Storek, Michael
    Wong, Nancy
    Patel, Parija
    Jiang, Xiaoyu
    Vagge, Deepthi S.
    Wardecki, Marek
    Shafer, Frank
    Lee, Michelle
    Broome, Catherine M.
    BLOOD, 2022, 140 (09) : 980 - 991
  • [42] Efficacy of Levofloxacin in the Treatment of BK Viremia: A Multicenter, Double-Blinded, Randomized, Placebo-Controlled Trial
    Lee, Belinda T.
    Gabardi, Steven
    Grafals, Monica
    Hofmann, R. Michael
    Akalin, Enver
    Aljanabi, Aws
    Mandelbrot, Didier A.
    Adey, Deborah B.
    Heher, Eliot
    Fan, Pang-Yen
    Conte, Sarah
    Dyer-Ward, Christine
    Chandraker, Anil
    CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2014, 9 (03): : 583 - 589
  • [43] Treatment options for nonalcoholic fatty liver disease: a double-blinded randomized placebo-controlled trial
    Anushiravani, Amir
    Haddadi, Niloufar
    Pourfarmanbar, Maedeh
    Mohammadkarimi, Vahid
    EUROPEAN JOURNAL OF GASTROENTEROLOGY & HEPATOLOGY, 2019, 31 (05) : 613 - 617
  • [44] A pilot trial for efficacy confirmation of 6′-sialyllactose supplementation in GNE myopathy: Randomized, placebo-controlled trial
    Park, Young-Eun
    Choi, Jaeil
    Kim, Lila
    Park, Eunjung
    Go, Hiroe
    Shin, Jinhong
    MOLECULAR GENETICS AND METABOLISM, 2025, 144 (01)
  • [45] Effect of Oral Lipid Matrix Supplement on Fat Absorption in Cystic Fibrosis: A Randomized Placebo-Controlled Trial
    Stallings, Virginia A.
    Schall, Joan I.
    Maqbool, Asim
    Mascarenhas, Maria R.
    Alshaikh, Belal N.
    Dougherty, Kelly A.
    Hommel, Kevin
    Ryan, Jamie
    Elci, Okan U.
    Shaw, Walter A.
    JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION, 2016, 63 (06): : 676 - 680
  • [46] Inhaled Nitric Oxide in Fibrotic Lung Disease A Randomized, Double-Blind, Placebo-controlled Trial
    Nathan, Steven D.
    Rajicic, Natasa
    Dudenhofer, Rosemarie
    Hussain, Rahat
    Argula, Rahul
    Bandyopadhyay, Debabrata
    Luckhardt, Tracy
    Muehlemann, Natalia
    Flaherty, Kevin R.
    Glassberg, Marilyn K.
    Lancaster, Lisa
    Raghu, Ganesh
    Fernandes, Peter
    ANNALS OF THE AMERICAN THORACIC SOCIETY, 2024, 21 (12) : 1661 - 1669
  • [47] A Randomized, Double-Blind, Placebo-Controlled Trial of Escitalopram in Patients with Asthma and Major Depressive Disorder
    Brown, E. Sherwood
    Sayed, Nasreen
    Van Enkevort, Erin
    Kulikova, Alexandra
    Nakamura, Alyson
    Khan, David A.
    Ivleva, Elena I.
    Sunderajan, Prabha
    Bender, Bruce G.
    Holmes, Traci
    JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE, 2018, 6 (05): : 1604 - 1612
  • [48] Effects of rotigotine on daytime symptoms in patients with primary restless legs syndrome: a randomized, placebo-controlled study
    Garcia-Borreguero, Diego
    Allen, Richard
    Hudson, John
    Dohin, Elisabeth
    Grieger, Frank
    Moran, Kimberly
    Schollmayer, Erwin
    Smit, Rene
    Winkelman, John
    CURRENT MEDICAL RESEARCH AND OPINION, 2016, 32 (01) : 77 - 85
  • [49] Placebo reactions in double-blind, placebo-controlled food challenges in children
    Vlieg-Boerstra, B. J.
    van der Heide, S.
    Bijleveld, C. M. A.
    Kukler, J.
    Duiverman, E. J.
    Dubois, A. E. J.
    ALLERGY, 2007, 62 (08) : 905 - 912
  • [50] Efficacy of high-flow oxygen therapy in all types of headache: a prospective, randomized, placebo-controlled trial
    Ozkurt, Birsen
    Cinar, Orhan
    Cevik, Erdem
    Acar, Ayhan Yahya
    Arslan, Deniz
    Eyi, Emrah Yusuf
    Jay, Loni
    Yamanel, Levent
    Madsen, Troy
    AMERICAN JOURNAL OF EMERGENCY MEDICINE, 2012, 30 (09): : 1760 - 1764