Browsing by Author "Richman, David P."

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    3,4-Diaminopyridine Base Effectively Treats the Weakness of Lambert-Eaton Myasthenia
    (Wiley, 2018) 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.; Neurology, School of Medicine
    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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    Reply: 3,4-diaminopyridine in LEMS: Concerns regarding presentation of previous studies
    (Wiley, 2018) Sanders, Donald B.; Harati, Yadollah; Juel, Vern C.; Lou, Jau-Shin; Marburger, Tessa; Pascuzzi, Robert M.; Peltier, Amanda C.; Richman, David P.; Smith, A. Gordon; Neurology, School of Medicine
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    Validation of the triple timed up‐and‐go test in Lambert‐Eaton myasthenia
    (Wiley, 2019-09) Raja, Shruti M.; Sanders, Donald B.; Juel, Vern C.; Harati, Yadollah; Smith, A. Gordon; Pascuzzi, Robert; Richman, David P.; Wu, Angie; Aleš, Kathy L.; Jacobus, Laura R.; Jacobus, David P.; Guptill, Jeffrey T.; Neurology, School of Medicine
    Introduction There are no validated, practical, and quantitative measures of disease severity in Lambert‐Eaton myasthenia (LEM). Methods Data from the Effectiveness of 3,4‐Diaminopyridine in Lambert‐Eaton Myasthenic Syndrome (DAPPER) trial were analyzed to assess triple timed up‐and‐go (3TUG) reproducibility and relationships between 3TUG times and other measures of LEM severity. Results The coverage probability technique showed ≥0.90 probability for an acceptable 3TUG difference of ≤0.2, indicating that it is reproducible in LEM patients. The correlation between 3TUG times and lower extremity function scores was significant in subjects who continued and in those who were withdrawn from 3,4‐diaminopyridine free base. Worsening patient‐reported Weakness Self‐Assessment Scale and Investigator Assessment of Treatment Effect scores corresponded with prolongation of 3TUG times. Discussion The 3TUG is reproducible, demonstrates construct validity for assessment of lower extremity function in LEM patients, and correlates with changes in patient and physician assessments. These findings, along with prior reliability studies, indicate 3TUG is a valid measure of disease severity in LEM.