3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia.

Journal Article (Journal Article)

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 < 0.0001). W-SAS similarly demonstrated an advantage for continuous treatment over placebo (P < 0.0001). Requirement for rescue and adverse events were more common in the placebo group. DISCUSSION: This trial provides significant evidence of efficacy of 3,4-DAP in the maintenance of strength in LEM. Muscle Nerve 57: 561-568, 2018.

Full Text

Duke Authors

Cited Authors

  • Sanders, DB; Juel, VC; Harati, Y; Smith, AG; Peltier, AC; Marburger, T; Lou, J-S; Pascuzzi, RM; Richman, DP; Xie, T; Demmel, V; Jacobus, LR; AleŇ°, KL; Jacobus, DP; Dapper Study Team,

Published Date

  • April 2018

Published In

Volume / Issue

  • 57 / 4

Start / End Page

  • 561 - 568

PubMed ID

  • 29280483

Pubmed Central ID

  • PMC5900968

Electronic International Standard Serial Number (EISSN)

  • 1097-4598

Digital Object Identifier (DOI)

  • 10.1002/mus.26052


  • eng

Conference Location

  • United States