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Dr. Krieger: 
Thank you so much for joining us today. My name is Ana Krieger. I'm a Professor of Clinical Medicine at Weill Cornell Medical College in New York City, and the Chief of the Division of Sleep Neurology. We're going to be discussing the Treatment Options for the Management of Narcolepsy today.

Narcolepsy is a chronic condition without a known cure that affects approximately 5 out of 10,000 people. Its predominant feature is daytime sleepiness, although patients with type 1 narcolepsy also have cataplexy. Given the importance of the symptoms, treatment is often recommended, and the approaches are focused on education about the disease and expectations during a life course, pharmacotherapy, and non-pharmacological approaches. Part of the screening as well is to evaluate patients for possible comorbid conditions, including obstructive sleep apnea, restless leg syndrome, mood disorders, and also diabetes. We need to remember that treatment is symptomatic, and not only pertaining to sleepiness, but as well as sleep fragmentation or other symptoms that the patients may present, including asleep inertia.

Non-pharmacological treatments are focused on establishing adequate sleep hygiene, keep a regular sleep-wake schedule, avoidance of alcohol or sedatives, keeping a healthy diet, and regular physical activity. Many patients may also benefit from strategic napping. It is important always to have a discussion about safety, including driving or operating equipment that requires sustained vigilance. Approaches that don't deal necessarily with medication are also especially important for the pediatric population and pregnant women, as often the decision may be made to stop medications during pregnancy.

In terms of pharmacotherapy, this slide illustrates the FDA approved medications to treat both excessive daytime sleepiness and cataplexy, which are often associated with narcolepsy. And the strength and the recommendation based by an international panel of specialists is also listed on this slide, which is important for people in making decisions from the healthcare providers’ perspective, determining which approaches to use for patients. The daytime use of wake-promoting agents and stimulants such as modafinil, armodafinil, solriamfetol, pitolisant, methylphenidate, and dextroamphetamine are important to consider.

However, there are also nighttime treatments that are important for the providers to understand how to use them, and they are all the different kinds of oxybates, which are also very effective in managing narcolepsy. We currently have three formulations of oxybates that require twice-a-night dose approved by the FDA. Those include sodium oxybate, a mixed salt oxybate, and a low salt oxybate. There is also a recent approval for a once-a-night dose of a sodium oxybate that is also available and approved by the FDA to treat both sleepiness and cataplexy associated with narcolepsy.

As with any other medication, when prescribing a pharmacological agent to patients, we need to make them aware of potential side effects. And they need to be carefully monitored in order to adjust the doses or change the approach. This is summarized on this particular slide, which reviews the potential adverse events and typical doses for those FDA approved medications. We need to remember that the medications have to be personalized and discussed with the patients, considering which agents would best fit their needs and most effectively improve their symptoms.

In terms of safety and clinical outcomes, a recent meta-analysis of the available studies compared safety outcomes and clinical improvement from several agents. And it was very important for us to see that the low sodium oxybate actually had the lowest risk for adverse events and led to the highest improvement in the clinical scale of patient outcomes. So, it's important for us clinically to understand how those medications work, how safe they are, and how best to apply them to a given patient. And remember that patients’ symptoms may change over time, their needs may change over time, so we should be able to adjust their treatment based on what is actually available.

Thank you so much for joining us.

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