Narcolepsy is a chronic sleep disorder for which there is no known cure. The onset of symptoms can begin at any age but frequently occurs during childhood or adolescence. This condition continues to impact patients throughout their lifetime. Management plans require implementation of ongoing pharmacological therapy to keep the symptoms under control for most patients, and patients may need substantial lifestyle adjustment such as maintaining nocturnal sleep hygiene and regular scheduling of daytime naps. Narcolepsy management plan development should consider balance between the tolerance to available medications and impact of certain comorbidities associated with the disorder.
In episode 1, listen as Sleep Review’s Sree Roy and neurologist-sleep specialist Michael Thorpy, MB, ChB, discuss:
Since narcolepsy starts at a young age and there is no cure as of yet, what are the long-term management implications?
What are the key symptoms of narcolepsy, and how and when do they typically manifest?
Do narcolepsy symptoms evolve over the course of a lifetime?
What can you typically accomplish with an individualized management plan?
How often do you reassess narcolepsy patients to determine whether their management plan needs to be adjusted?
Are there specific guidelines that you recommend to the physicians in the audience with regard to managing narcolepsy
Sree Roy: Hello and welcome. I’m Sree Roy with Sleep Review, and I’m thrilled to be here with neurologist-sleep specialist Michael Thorpy, MBChB. Dr. Thorpy is director of the Sleep-Wake Disorders Center at Montefiore Medical Center in the Bronx, New York.
This episode is sponsored by Jazz pharmaceuticals.
Today, we are chatting about the lifelong burden of narcolepsy. Narcolepsy onset occurs during childhood or adolescence, typically. Since it starts at a young age and there is no cure as of yet, what are the long term management implications?
Dr. Michael J. Thorpy: Well, that’s a disorder that as you mentioned, begins in childhood. And so children have symptoms that are typically excessive daytime sleepiness, and then there’s this abnormal muscle activity that we call cataplexy
This can be very disabling for a child and so it can affect their education. And, then of course, as children get older, it interferes with their personal relationships. So it can affect not only family relationships, but also relationships with others. And so this may be a very negative effect as they get to early adulthood and meeting prospective partners.
And, of course, it can affect their education, college education, their work as they enter into the work time of life. And maybe a major factor that affects things such as driving or any dangerous activities that are a risk.
And it persists right through to old age. And so elderly people find that it interferes with their retirement time because they’re tired and sleepy and unable to do the things that they want to do in their retirement. So it affects people right across the whole life.
Sree Roy: You touched on this a little bit, but could you explain what are the key symptoms of narcolepsy and how, and when do they typically manifest?
Dr. Michael J. Thorpy: Yep. Well, narcolepsy is a disorder of excessive daytime sleepiness, so it’s a chronic neurological disorder that causes patients to be tired, fatigued, and sleepy throughout the day so that they can fall asleep at inappropriate times.
They may have episodes of sleep attacks where they’re doing things and they’re unable to resist the sleepiness that comes over them and they have to give into it. But in all patients, there’s a background level of sleepiness that’s there the whole time that affects their cognitive function. And typically this begins in children and persists and may get more severe as time goes on. And then in addition to the sleepiness, there is a unusual symptom that we call cataplexy, which is an emotionally induced muscle weakness, so that when the person becomes emotional and maybe laughing or hearing a joke or sudden surprise or anxiety, they will get a weakness that comes over them. At its worst, that can affect their whole body to the point that they will actually fall to the ground.
But for many patients, it affects either their head, their neck, a feeling of weakness in their legs, or they may have weakness in their arms and drop things. And it’s a very disabling symptom because it can come on very suddenly and when they’re not expecting it and they find it extremely embarrassing and potentially can be dangerous too, if they fall and or if they’re driving at the time when this type of muscle weakness occurs.
So there’s the sleepiness, there’s the cataplexy, but also these patients have disturbed, nocturnal sleep. So their sleep at night is not good. In fact, for patients with narcolepsy have difficulty staying awake during the day, but they have difficulty in sleeping at night and they have a lot of interruptions to their sleep, and they have a lot of disrupted dream sleep activity so that they may have nightmares, fragments of dreams, hallucinations, partial manifestations of muscle weakness. We call sleep paralysis. There’s a lot of disturbed nocturnal sleep that goes along with the sleepiness and the emotionally induced muscle weakness, the cataplexy.
Sree Roy: Do narcolepsy symptoms evolve over the course of a lifetime? If so, how? What patterns do they follow?
Dr. Michael J. Thorpy: Well, very often the symptoms are worse at the onset. Although, we know that the sleepiness is the first symptom that occurs and patients may develop the cataplexy, the emotionally induced muscle weakness of varying times after the onset of the sleepiness.
But very often for most patients, because the symptoms are profound at the beginning, they learn to adapt their life accordingly. So they will learn to take a nap before they need to do something important, or they learn to avoid emotionally inducing situations. So for most patients, they find that the symptoms actually do improve a little bit as time goes on, but they never go away for patients with narcolepsy it’s a lifelong disorder. So there’re always some symptoms that are present.
Sree Roy: Let’s talk about alleviating the clinical burden for people with narcolepsy as much as possible. What can you typically accomplish with an individualized management plan? For example, do you set specific goals such as work or school productivity and/or improvements in quality of life?
Dr. Michael J. Thorpy: Yeah, well, quality of life is extremely important for these patients. And so we need to discuss that and with their management, because although we have medications that can help patients, they are not able to return the patient to normal. So patients will always have some symptoms despite optimal treatment with medications. So we need to discuss that with patients as to how they’re doing with it, whether there’s resolution of the cataplexy, for example. And the cataplexy tends to be a little bit easier to resolve, but we may need to have multiple treatment options tried in a particular patient in order to get that cataplexy under control.
The sleepiness is much more difficult. And so patients tend to have ongoing sleepiness, even though it may be improved with management by medications. But they need to understand that it is going to be present to some extent, and they need to adapt their life accordingly so that certain occupations are not ideal for a patient.
For example, sitting in front of a computer all day is not good because it’s very soporific and patients will tend to be more tired and fatigued and sleepy in that situation. So more active activities are important. So discussing the patient’s goals in life and their educational needs is very important when managing narcolepsy.
Sree Roy: We’ll be back with Dr. Thorpy after a short break.
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Sree Roy: How often do you generally reassess narcolepsy patients to determine whether their management plan needs to be adjusted?
Dr. Michael J. Thorpy: When we first see patients with narcolepsy and we try various medications, the management can be quite difficult because unfortunately, many of the medications that we use tend to have adverse effects and patients with narcolepsy do have underlying comorbidities, psychiatric and medical comorbidities.
For example, anxiety and depressive features are very common in narcolepsy, metabolic disorders and cardiovascular disorders also occur in patients with narcolepsy. So the medications that we use can interact and interfere with those other comorbidities. And so often with patients, it’s a matter of finding the best medication for them. And so we may need to try multiple medications and many times we need to use polypharmacy. So I would say the majority of patients with are on two medications and some patients need to take three medications in a day.
But getting to that point where a patient is relatively stable does take some time. And so we see the patient very frequently in the early stages of getting them adjusted to medication, but then once they’re stabilized, we can see them at less frequent intervals, for example, every six months or 12 months. But there’s always issues that come up. And even though a patient may be stable on a medication, it doesn’t prevent them from subsequently developing adverse effects. So we do need to keep in close contact with patients to monitor their progress and how effective the medications are and change the medications, either the dosing of the medications or change to a different medication as the need arises.
Sree Roy: Are there specific guidelines that you recommend to the physicians in the audience with regard to managing narcolepsy?
Dr. Michael J. Thorpy: Well, there are guidelines that have been produced in the United States by the American Academy of Sleep Medicine. These guidelines are really about the medications and their effectiveness in treating narcolepsy. They’re not really a treatment algorithm. So you need to know which medications are the ones that are most effective for one or more of the symptoms. As I mentioned, the sleepiness, some medications are more effective than others. Some medications will treat both the sleepiness and the cataplexy. And this is all detailed in the American Academy of Sleep Medicine practice parameters.
There are also guidelines that have been produced in Europe by the European Narcolepsy Societies. And these guidelines are more of a treatment algorithm. So they give you a better idea as to if when medication doesn’t work, you could move to another medication and what would be appropriate for a particular patient.
But for most patients, treatment does need to be individualized. No two patients with narcolepsy are the same. Very often it’s a matter of trial and error with medications to see what’s going to be the most effective for them and with the least side effects.
Sree Roy: Thank you so much for chatting with us about narcolepsy today. You can find Sleep Review at sleepreviewmag.com. And thank you to our audience for tuning into this episode.
To dive even deeper:
Thorpy MJ, Hiller G. The medical and economic burden of narcolepsy: implications for managed care. Am Health Drug Benefits. 2017;10(5):233-241.
Thorpy M, Morse AM. Reducing the clinical and socioeconomic burden of narcolepsy by earlier diagnosis and effective treatment. Sleep Med Clin. 2017;12(1):61-71.
Morse AM. Narcolepsy in children and adults: a guide to improved recognition, diagnosis and management. Med Sci (Basel). 2019;7(12):E106.
de Biase S, Gigli GL, Valente M. Important decisions in choosing the pharmacotherapy for narcoleptics. Expert Opin Pharmacother. 2019;20(5):483-486.
Wozniak DR, Quinnell TG. Unmet needs of patients with narcolepsy: perspectives on emerging treatment options. Nat Sci Sleep. 2015;7:51-61.
Abad VC, Guilleminault C. New developments in the management of narcolepsy. Nat Sci Sleep. 2017;9:39-57.