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Dr. Morse:
This is CME on ReachMD, and I'm Dr. Anne Marie Morse. We're going to discuss the evolving therapies for pediatric narcolepsy in this episode.
We know that twice-nightly oxybates have been available for quite the past few years, and that's on the background of over 25 years of experience utilizing oxybate therapies for treatment that really can be pivotal in terms of improving not only excessive daytime sleepiness, but also cataplexy. We recognize that the way this medication works is really quite unique. Although many will argue that we don't fully understand the mechanism of action, there's lots of literature that supports how it works.
One of the things that I do find interesting, however, is this is a medication where its indications are for the daytime; however, that's when the medication’s out of your system, so clearly having quite a remarkable impact on the brain's function.
Now, one of the challenges that I've experienced as a physician taking care of children and adolescents with narcolepsy and utilizing twice-nightly oxybates, is the burden that it can sometimes cause for not only the child but the entire family with a forced awakening in the middle of the night. With a once-nightly oxybate that currently is FDA approved in adults 18 and older, I've had the privilege in my own clinical practice to be able to utilize this in some children. We currently know that once-nightly sodium oxybate is waiting for its PDUFA in September for potentially getting the approval and indication in pediatric narcolepsy.
What has been my experience with the use of one-nightly sodium oxybate in children and adolescents? Well, needless to say, it's been quite remarkable. The children that I've had who've either been naive to sodium oxybate or have been a transition from twice-nightly sodium oxybate, but were struggling with getting up for that second dose, have successfully either transitioned or initiated with a once-nightly sodium oxybate.
The reality is that with a growing spectrum of oxybates available, including twice-nightly sodium oxybate, twice-nightly mixed salt oxybate, and a once-nightly sodium oxybate, it now is expanding my ability to be able to reach for a medication that really reaches and treats all the symptoms of narcolepsy.
Although the FDA indication is for excessive daytime sleepiness and cataplexy, the ability to provide a more consolidated sleep with less REM intrusion allows me also to get at some of those other pentad symptoms, like sleep-related hallucinations, sleep paralysis and, of course, disturbed nocturnal sleep. In addition to that, some of the things that maybe you're not thinking about or potentially even asking your patients about, like nightmare disorder, many times are also improved. It's always important, when talking about oxybate medications, to make sure that you're really giving a level playing ground in regards to, not only what are the potential benefits you may experience, but also the potential risks?
We definitely always will talk about the fact that it's a CNS depressant and that if there is pre-existing sleep disordered breathing, we're going to want to monitor that more closely to ensure that that doesn't necessarily worsen or have treatment-emergent symptoms of sleep disordered breathing, such as obstructive or central sleep apnea. These are all things that you're going to want to personalize and attend to an individual basis.
No matter what medication I'm reaching for in my clinical practice, I also always make sure to discuss the fact that my intention is to treat your narcolepsy, and with that, I need to cross the blood-brain barrier and act on your brain. And when I'm acting on your brain, I'm sharing that organ with another specialty, and that’s psychiatry. And so whether there's pre-existing psychological or psychiatric diagnoses or not, I'm going to discuss safety plans as well as how we're going to monitor mood and behavior. Making sure we're proactive rather than reactive, adds an additional layer of security, no matter what medication I'm utilizing.
So one of the things that I'm looking forward to is seeing when the PDUFA date comes in September, and how we'll be able to personalize the use of another treatment that is only going to further improve our ability to treat all of our children who have narcolepsy.
Well, this was a great but brief discussion. Hopefully you can put some of these tips into your own practice tomorrow, and maybe even in a few months, if we see the treatment landscape changing. And thank you again for listening.