Premium
3,4‐diaminopyridine base effectively treats the weakness of Lambert‐Eaton myasthenia
Author(s) -
Sanders Donald B.,
Juel Vern C.,
Harati Yadollah,
Smith A. Gordon,
Peltier Amanda C.,
Marburger Tessa,
Lou JauShin,
Pascuzzi Robert M.,
Richman David P.,
Xie Tai,
Demmel Valentin,
Jacobus Laura R.,
Aleš Kathy L.,
Jacobus David P.
Publication year - 2018
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26052
Subject(s) - placebo , myasthenia gravis , lambert eaton myasthenic syndrome , medicine , clinical endpoint , adverse effect , weakness , anesthesia , randomized controlled trial , double blind , surgery , alternative medicine , pathology
: 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