The expected benefit of amifampridine is better muscle strength and day-to-day function.
“Many people have improvements in muscle strength, walking ability, stamina, and performance of everyday activities,” AlQahtani says, adding that some people notice improvement in symptoms such as dry mouth.
Amifampridine has also been shown to increase muscle strength, and improvement in autonomic symptoms has been shown in placebo-controlled clinical trials.
Research suggests that amifampridine may help with forced vital capacity, a measure of lung function, and help people lower their corticosteroid dose, too.
If symptoms aren’t well managed or remain bothersome, the first step is usually making sure that the dose has been optimized within recommended safety limits, says AlQahtani.
If amifampridine is not tolerated or not available, pyridostigmine (Mestinon) may be considered, and it can also be added to amifampridine for extra symptom relief.
“Pyridostigmine slows the breakdown of acetylcholine at the neuromuscular junction, which can leave more of that chemical messenger available to help muscles contract,” says AlQahtani.
If symptom control is still not enough, doctors may consider treatments that target the autoimmune process behind LEMS. “Options may include intravenous immunoglobulin (IVIG), corticosteroids, azathioprine, mycophenolate mofetil, and rituximab, depending on disease severity, other health conditions, and patient-specific factors,” says AlQahtani.
When LEMS is associated with cancer, treating the cancer is a central part of therapy and may also improve LEMS symptoms, he says.
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