1. 3,4‐diaminopyridine base effectively treats the weakness of Lambert‐Eaton myasthenia
- Author
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Sanders, Donald B, Juel, Vern C, Harati, Yadollah, Smith, A Gordon, Peltier, Amanda C, Marburger, Tessa, Lou, Jau‐Shin, Pascuzzi, Robert M, Richman, David P, Xie, Tai, Demmel, Valentin, Jacobus, Laura R, Aleš, Kathy L, Jacobus, David P, and Team, The Dapper Study
- Subjects
Biomedical and Clinical Sciences ,Clinical Sciences ,Clinical Trials and Supportive Activities ,Clinical Research ,Evaluation of treatments and therapeutic interventions ,6.1 Pharmaceuticals ,Adult ,Aged ,Aged ,80 and over ,Amifampridine ,Deprescriptions ,Double-Blind Method ,Female ,Humans ,Lambert-Eaton Myasthenic Syndrome ,Maintenance Chemotherapy ,Male ,Middle Aged ,Muscle Weakness ,Neuromuscular Agents ,Young Adult ,3 ,4-diaminopyridine ,amifampridine ,clinical trial ,Eaton-Lambert syndrome ,efficacy ,ELS ,Lambert-Eaton myasthenia ,Lambert-Eaton myasthenic syndrome ,Lambert-Eaton syndrome ,LEMS ,LES ,timed up-and-go ,Dapper Study Team ,3 ,4-diaminopyridine ,Medical and Health Sciences ,Neurology & Neurosurgery ,Biological sciences ,Biomedical and clinical sciences - Abstract
INTRODUCTION:3,4-diaminopyridine has been used to treat Lambert-Eaton myasthenia (LEM) for 30 years despite the lack of conclusive evidence of efficacy. METHODS:We conducted a randomized double-blind placebo-controlled withdrawal study in patients with LEM who had been on stable regimens of 3,4-diaminopyridine base (3,4-DAP) for ≥ 3 months. The primary efficacy endpoint was >30% deterioration in triple timed up-and-go (3TUG) times during tapered drug withdrawal. The secondary endpoint was self-assessment of LEM-related weakness (W-SAS). RESULTS:Thirty-two participants were randomized to continuous 3,4-DAP or placebo groups. None of the 14 participants who received continuous 3,4-DAP had > 30% deterioration in 3TUG time versus 72% of the 18 who tapered to placebo (P
- Published
- 2018