Primary respiratory failure as the presenting symptom in Lambert–Eaton myasthenic syndrome

Barr, C W; Claussen, Gwen C.; Thomas, D. J.; Fesenmeier, James T.; Pearlman, Ross L; Oh, Shin J.

doi:10.1002/mus.880160704

Cited by 25 publications

(1 citation statement)

References 7 publications

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“…This is not expected in view of rare occurrence of breathing difficulty in LEMS. 26 This most likely represents an improvement in chest muscle function with amifampridine phosphate.…”

Section: Discussionmentioning

confidence: 98%

Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS

Shieh

Sharma

Kohrman

et al. 2019

Journal of Clinical Neuromuscular Disease

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Objective: To assess tolerability and efficacy of amifampridine phosphate versus placebo for symptomatic treatment of Lambert–Eaton Myasthenic Syndrome (LEMS). Methods: This phase 3 randomized, double-blind, placebo-controlled withdrawal trial in 26 adults with LEMS compared efficacy of amifampridine phosphate versus placebo over a 4-day period. The primary endpoints were quantitative myasthenia gravis score (QMG) and subject global impression, and the secondary endpoint was Clinical Global Impression–Improvement. The exploratory endpoints were 3TUG (timed up and go) test and QMG limb domain score. All participants had been receiving amifampridine phosphate (30–80 mg/d divided into 3 or 4 doses daily) in an expanded access protocol and had been titrated to the optimal dose and frequency for at least 1 week before randomization into the current study. After completion of assessments after 4 days of double-blind treatment, patients had the option to return to open-label amifampridine phosphate. The efficacy endpoints were mean changes from baseline in the various evaluation parameters. Results: Amifampridine phosphate (n = 13) demonstrated significant benefit in QMG and subject global impression compared with placebo (n = 13) at 4 days. Other measures of efficacy, including Clinical Global Impression–Improvement, 3TUG, and QMG limb domain score also improved. The most common “adverse events” in the placebo group were muscle weakness (n = 5) and fatigue (n = 4), as expected from withdrawal of amifampridine phosphate, whereas only back pain (n = 1), pain in extremity (n = 1), and headache (n = 1) were reported in amifampridine phosphate group. Conclusions: This phase 3 randomized, double-blind, placebo-controlled withdrawal trial in adults with LEMS provided class I evidence of efficacy of amifampridine phosphate as symptomatic treatment in LEMS.

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“…This is not expected in view of rare occurrence of breathing difficulty in LEMS. 26 This most likely represents an improvement in chest muscle function with amifampridine phosphate.…”

Section: Discussionmentioning

confidence: 98%

Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS

Shieh

Sharma

Kohrman

et al. 2019

Journal of Clinical Neuromuscular Disease

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Lambert-Eaton myasthenic syndrome presenting with severe respiratory failure

et al. 1996

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Two cases of Lambert‐Eaton myasthenic syndrome (LEMS) who presented with primary respiratory failure are reported. In each case, although not initially suspected clinically, the electrophysiological findings, which included reduced compound muscle action potential amplitudes, decrement to 3‐Hz stimulation, and potentiation after 40‐Hz stimulation, led to the diagnosis in the critical care unit. Electrophysiological studies of the respiratory system, including repetitive nerve stimulation of the phrenic nerve, were extremely valuable in management. As shown by these cases, the severe respiratory failure in LEMS is reversible with treatment. Thus, LEMS should be considered in cases of unexplained respiratory failure, other clinical features of the disorder sought, and the electrophysiological hallmarks looked for, including studies of the respiratory system. © 1996 John Wiley & Sons, Inc.

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Low-dose guanidine and pyridostigmine: relatively safe and effective long-term symptomatic therapy in Lambert-Eaton myasthenic syndrome

Kim

Head

et al. 1997

Muscle Nerve

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Primary respiratory failure as the presenting symptom in Lambert–Eaton myasthenic syndrome

Cited by 25 publications

References 7 publications

Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS

Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS

Lambert-Eaton myasthenic syndrome presenting with severe respiratory failure

Low-dose guanidine and pyridostigmine: relatively safe and effective long-term symptomatic therapy in Lambert-Eaton myasthenic syndrome

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