Pediatric Narcolepsy: How to Address Its Burden of Illness Through Prompt Recognition, Diagnosis, and Management

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Dr. Brown:
Hi everyone, this is CME on ReachMD and I'm Dr. Brown. And today we're gonna be talking about the evolving therapies for managing pediatric narcolepsy. So when it comes to a treatment overview, really there are three main key elements. There are elements such as the medication management, but there's also sleep hygiene and healthy sleep habits, as well as safety and lifestyle factors. And one thing that I usually encourage is that you target the most bothersome symptoms. So there are three main goals. The goal is to improve the quality and duration of daytime wakefulness and performance, knowing that excessive daytime sleepiness may not be completely eliminated, but at least making sure that we control it as much as possible.

And also achieving elimination of the hallucinations and cataplexy symptoms that patients have. Now with pharmacologic management, the various treatment modalities available when we're talking about excessive sleepiness, some of those treatment options include modafinil or modafinil methylphenidates, dextroamphetamine, oxidates, sofe patan. When we're talking about addressing the cataplexy or REM intrusion phenomena, usually we're talking about medications like venlafaxine, sodium oxybate again, and Pitocin. One general thing to be aware of is usually you might need two or more medications to control these symptoms. Some of the medications have been approved for children over seven years of age, for instance, the oxybate. And there are two forms of oxybate, and I'm just gonna mention that briefly. There are the twice nightly dosing and then there's also the one nightly dosing available. Now, additional medication considerations include knowing that the goal of one medication may be to achieve multiple functions.

So you might have a medication, for instance, again, like the oxybate that address excessive day and sleepiness, but also help treat the cataplexy or medications like petant, which also ends up addressing the excessive daytime sleepiness and also addressing the cataplexy. So it is a histamine inverse agonist and what it does is that it enhances the histamine heric neurotransmission in the brain and it promotes wakefulness. Again, improves excessive daytime sleepiness. Usually the dosing is between nine and 36 milligrams titrated weekly till you reach your goal. And really the good thing about this medication is that it has no true effect on sleep architecture. There are, however, some side effects like headaches, nausea, insomnia, and irritability. There's been some, you know, potential possibility of it interacting with birth control. Now, when it comes to using petant, it was initially approved for adults.

Recently there was a PDUFA meeting to really talk about its role in the pediatric age group. But I wanna reference one study that was published in 2023. And this study looked at the effect of petant in children. And so they had about 115 participants aged between six to 17 years with narcolepsy, with and without cataplexy. And they compared the use of pitolisant versus placebo and they assessed their excessive data and sleepiness using the Ullanlinna Narcolepsy Scale or UNS. It's a little bit different than your airport sleepiness scale because it actually measures cataplexy as well. And what were the results? They found out that there was significant improvement in excessive daytime sleepiness and overall sleep quality using the UNS scale. And one other thing they noticed was that the safety profile was similar to adults, both of which are reassuring. Now, a little bit about non-medical treatments include things like ensuring education of the patients, their families and schools. You need to help them reestablish realistic goals and performance standards. For instance, they may need modified homework assessments. They may need extra time for test taking. They may need supervision, especially around activities like swimming or driving and cooking. You wanna avoid, dealing with heavy machinery. You also want to instruct them not to drive while sleepy. And one of the things I typically recommend strongly is that we have these patients within support groups or their family members within support groups. Some of the well-known support groups include things like the narcolepsy network, wake up narcolepsy, as well as project sleep. Now this was another micro discussion on this very important topic, but our time is up. And thank you for listening.

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