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

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 < 

SUBMITTER: Sanders DB 

PROVIDER: S-EPMC5900968 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature