La 3,4-diaminopyridine base traite efficacement la faiblesse de la myasthénie de Lambert-Eaton.

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La 3,4-diaminopyridine base traite efficacement la faiblesse de la myasthénie de Lambert-Eaton.

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:arrow: Lu sur :https://www.ncbi.nlm.nih.gov/pubmed/29280483

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3,4-Diaminopyridine Base Effectively Treats the Weakness of Lambert-Eaton Myasthenia.
Sanders DB1, Juel VC1, Harati Y2, Smith AG3, Peltier AC4, Marburger T5, Lou JS6, Pascuzzi RM7, Richman DP8, Xie T9, Demmel V10, Jacobus LR11, Aleš KL11, Jacobus DP11; DAPPER Study Team.
Muscle Nerve. 2017 Dec 27. doi: 10.1002/mus.26052.


Abstract

  • INTRODUCTION:

    3,4-diaminopyridine has been used to treat Lambert Eaton myasthenia (LEM) for thirty years despite the lack of conclusive evidence of efficacy.
  • METHODS:

    We conducted a randomized double-blind placebo-controlled withdrawal study in LEM patients 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:

    32 participants were randomized to continuous 3,4-DAP or placebo. None of the 14 receiving continuous 3,4-DAP had >30% deterioration in 3TUG time vs 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). Need 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.

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