CATNAP® registry: collecting real-world data to enhance understanding of paediatric narcolepsy
Paediatric narcolepsy is a debilitating and life-long condition. Currently, there is limited data on its natural history, symptoms and management. The Children, Adolescents and their providers: The Narcolepsy Assessment Partnership (CATNAP®) registry, supported by Jazz Pharmaceuticals as the initial sponsor, aims to collate real-world data from patients, caregivers and physicians to give insight into the condition and improve its diagnosis, treatment outcomes and management
What is Narcolepsy?
Narcolepsy is a chronic neurological condition, which affects nerve cells and chemicals in the brain. It is a rare condition that is estimated to affect 1 in 2000 people.1 Having narcolepsy means the brain is unable to regulate normal sleeping and waking patterns. Those with the condition can feel excessively tired during the day and can fall asleep at inappropriate times, often while doing everyday activities. Symptoms generally appear in childhood and early adolescence but are often not diagnosed as symptoms of narcolepsy until adulthood. Symptoms can differ depending on the person’s age at symptom onset and may vary over time in frequency and intensity.
The symptoms of narcolepsy are:
- excessive daytime sleepiness – feeling very drowsy during the day and finding it hard to stay awake, often falling asleep suddenly and without warning
- cataplexy – partial or total loss of muscle control when awake, often triggered by strong emotions such as laughter or anger
- sleep paralysis – a temporary inability to move or speak when waking or falling asleep
- hallucinations – excessive dream-like perceptions when falling asleep or waking up
Having narcolepsy can have a significant impact on a person’s life. It can result in difficulty with everyday tasks and can affect someone’s home life and school or work. It can be hard to cope with emotionally and the symptoms can be frightening and difficult to manage, especially for children.
Diagnosing paediatric narcolepsy can be challenging. Symptoms usually present between the ages of 10 and 25, but if symptoms start before the age of 18, they are more likely to be missed, resulting in some children not receiving a diagnosis until adulthood. Therefore, it is vital to raise awareness and promote better understanding among parents, children and adolescents, school professionals, the public and even paediatric healthcare providers.
Factors that can make diagnosing paediatric narcolepsy difficult include:
- varying sleep patterns and duration of sleep in children
- the difficulty for children to communicate their symptoms
- differing symptoms of cataplexy in children
- misinterpretation of the symptoms by caregivers or clinicians
- misdiagnosis of narcolepsy symptoms; for example, excessive tiredness is often diagnosed as chronic fatigue and hallucinations can be mistaken as symptoms of psychosis or epilepsy
Unfortunately, narcolepsy symptoms in children and adolescents are often mistaken for laziness and disinterest.
The need for real-world evidence
Narcolepsy can drastically affect all aspects of a child’s life. It can affect their performance at school, participation in play and sports as well as their ability to sustain friendships. It can have an impact on their mental health, their independence, their confidence and self-esteem. However, there is currently a lack of data to accurately measure how these issues may affect a child throughout their life. Unfortunately, existing narcolepsy registries do not focus on paediatric narcolepsy and do not use data that is input directly by the child, or by their parent or guardian.
Collecting observational real-world data from participants on the effects of narcolepsy on daily life will be invaluable in improving the quality of life for people with the condition and advancing medical treatments. It will also provide natural history data, which is currently hard to establish because of the challenges in making a timely diagnosis.
Children, Adolescents and their providers: The Narcolepsy Assessment Partnership (CATNAP)®
The CATNAP® registry aims to fulfil these unmet data needs. The web-based registry focuses on the health and quality of life of under-18s with narcolepsy in the US. The registry platform provides a patient and a clinician/researcher user interface to capture the relevant data and provides collaborative environment to share registry insights and educational information. The data captured in the registry encompasses profile, demographics, medical and treatment history and quality of life. The collected data will provide valuable scientific information and insight to the study of paediatric narcolepsy.
The registry aims to:
- help researchers and physicians learn more about paediatric narcolepsy to improve treatments, outcomes and quality of life for patients and their families
- better inform the best pathways for the management of narcolepsy
- help patients, physicians and researchers to better understand the natural course of the condition
- understand the daily impact of narcolepsy on patients and caregivers
- improve the time to diagnosis for patients
- inform treatment guidelines for professional organizations
Insights so far
Enrolment to the registry began in October 2020. Since then, the registry has already garnered key insights on paediatric narcolepsy.
The following common symptoms were reported at diagnosis: 96% excessive daytime sleepiness, 64% cataplexy, 64% disrupted sleep, 52% vivid dreams and 40% nightmares.
The most common psychiatric comorbidities reported: 20% anxiety, 16% depression, 12% attention deficit/hyperactivity disorder (ADHD), 8% panic attacks.
As the registry collects more real-world experience and data from patients, caregivers and clinicians, researchers and physicians will be able to improve and enhance their understanding of paediatric narcolepsy. Not only will they better understand the manifestations and management of the condition, but they will also gain insight into the burden of the condition and its impact on patients and their families.
“CATNAP offers a unique opportunity to longitudinally study symptom burden, treatment response, and prognosis of children and young adults with narcolepsy. These data are needed to further our understanding of pediatric narcolepsy and spur research and drug development to improve disease burden.”
Dr. Kiran Maski, MD, MPH at Boston Children’s Hospital
This comprehensive data will serve to advance study into improving treatments and outcomes; educate and inform patients, the public and healthcare professionals; improve the time to diagnosis; and inform clinical decision making. All with the aim of ultimately improving the lives of those living with narcolepsy.
About Pulse Infoframe
The CATNAP® registry is managed and powered by Pulse Infoframe. Pulse is a real-world evidence generation, health informatics and insights company that provides a technology and services platform designed to extract, curate, analyze and disseminate evidence-based conclusions that improve the quality of people’s lives. Learn more at www.pulseinfoframe.com
Children, Adolescents, and Their providers: the Narcolepsy Assessment Partnership (CATNAP™) Paediatric Narcolepsy Registry—Baseline Demographics
Children, Adolescents, and Their providers: the Narcolepsy Assessment Partnership (CATNAP™) Paediatric Narcolepsy Registry: Study Design