Clinical Characteristics and Burden of Illness in Pediatric Patients with Narcolepsy

Plazzi, Giuseppe; Clawges, Heather M.; Owens, Judith

doi:10.1016/j.pediatrneurol.2018.06.008

Cited by 88 publications

(81 citation statements)

References 92 publications

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“…90 Most children with narcolepsy are brought to their physicians’ attention because of their excessive daytime sleepiness, similar to adults. 91 Some children may restart taking naps after having given them up. The naps may not be refreshing, which may differ from adults, who often characterize their naps as refreshing.…”

Section: Challenges and Prospects For Specific Populations With Narcomentioning

confidence: 99%

<p>Living with Narcolepsy: Current Management Strategies, Future Prospects, and Overlooked Real-Life Concerns</p>

Barker¹,

Flygare²,

Paruthi³

et al. 2020

NSS

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Narcolepsy is a neurological disorder of the sleep-wake cycle characterized by excessive daytime sleepiness (EDS), cataplexy, nighttime sleep disturbances, and REM-sleep-related phenomena (sleep paralysis, hallucinations) that intrude into wakefulness. Dysfunction of the hypocretin/orexin system has been implicated as the underlying cause of narcolepsy with cataplexy. In most people with narcolepsy, symptom onset occurs between the ages of 10 and 35 years, but because the disorder is underrecognized and testing is complex, delays in diagnosis and treatment are common. Narcolepsy is treated with a combination of lifestyle modifications and medications that promote wakefulness and suppress cataplexy. Treatments are often effective in improving daytime functioning for individuals with narcolepsy, but side effects and/or lack of efficacy can result in suboptimal management of symptoms and, in many cases, significant residual impairment. Additionally, the psychosocial ramifications of narcolepsy are often neglected. Recently two new pharmacologic treatment options, solriamfetol and pitolisant, have been approved for adults, and the indication for sodium oxybate in narcolepsy has been expanded to include children. In recent years, there has been an uptick in patient-centered research, and promising new diagnostic and therapeutic options are in development. This paper summarizes current and prospective pharmacological therapies for treating both EDS and cataplexy, discusses concerns specific to children and reproductive-age women with narcolepsy, and reviews the negative impact of health-related stigma and efforts to address narcolepsy stigma.

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Section: Challenges and Prospects For Specific Populations With Narcomentioning

confidence: 99%

<p>Living with Narcolepsy: Current Management Strategies, Future Prospects, and Overlooked Real-Life Concerns</p>

Barker¹,

Flygare²,

Paruthi³

et al. 2020

NSS

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“…Although a reduction of symptoms has been demonstrated (EDS and cataplexy) in 88% of NT1 children [78,79], sodium oxybate treatment maintains a high risk ratio: sleep walking, sleep enuresis, exacerbation of sleep apnea, tremor, constipation, exacerbation of pre-existing depressive tendencies, weight loss, nausea, irritability, and episodes of sleep drunkenness. Treatment cessation generally does not have rebound effects [77].…”

Section: Sodium Oxybate or Gamma Hydroxybutyratementioning

confidence: 99%

Narcolepsy treatment: pharmacological and behavioral strategies in adults and children

et al. 2019

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Narcolepsy is a disabling, rare, and chronic sleep disorder, currently classified as distinct central nervous system hypersomnia in narcolepsy type 1 (NT1) and narcolepsy type 2 (NT2). Although today a reliable pathogenic hypothesis identifies the cause of NT1 as an autoimmune process destroying hypocretin-producing cells, there is no cure for narcolepsy, and the symptomatic pharmacological available treatments are not entirely effective for all symptoms. Behavioral therapies play a synergistic role in the disease treatment. We here review the available therapeutic options for narcolepsy, including symptomatic pharmacological treatments as well as behavioral and psychosocial interventions that could help clinicians improve the quality of life of patients with narcolepsy in adulthood and childhood.

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“…However, patients often experience an extended diagnostic delay (14 years in Europe) [4] that may be associated with misdiagnosis and inappropriate resource utilization, increasing the psycho-social impact of the disease [3,5]. Quality of life and social activities are profoundly impaired in both adults and children [6,7], and patients need frequent and timely counselling to adjust behavioural and pharmacological treatments. High direct and indirect disease-related costs are reported [8][9][10].…”

Section: Introductionmentioning

confidence: 99%

Protocols of a diagnostic study and a randomized controlled non-inferiority trial comparing televisits vs standard in-person outpatient visits for narcolepsy diagnosis and care: TElemedicine for NARcolepsy (TENAR)

et al. 2020

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Background: Narcolepsy is a rare chronic sleep disorder that typically begins in youth. Excessive daytime sleepiness is the main disabling symptom, but the disease is often associated with severe endocrine-metabolic and psychosocial issues, worsened by a long diagnostic delay, requiring a multidisciplinary approach. The scarcity of reference Sleep Centres forces the patient and family to travel for seeking medical consultations, increasing the economic and psychosocial burden of the disease. Growing evidence suggests that Telemedicine may facilitate patient access to sleep consultations and its non-inferiority in terms of patient satisfaction, adherence to treatment, and symptom improvement for sleep disorders. However, Telemedicine clinical and economic benefits for patients with narcolepsy are still unknown. Methods: TENAR is a two-part project, including: 1. a cross-sectional study (involving 250 children and adults with suspected narcolepsy) evaluating the accuracy of Teletriage (i.e., a synchronous live interactive sleep assessment through a Televisit) for narcolepsy diagnosis compared to the reference standard; and 2. a twoarm, parallel, open randomized controlled trial (RCT) to demonstrate the non-inferiority of the multidisciplinary care of narcolepsy through Televisits versus standard care. In this RCT, 202 adolescents (> 14 y.o.) and adults with narcolepsy will be randomly allocated (1:1 ratio) either to Televisits via videoconference or to standard in-person outpatient follow-up visits (control arm). The primary outcome is sleepiness control (according to the Epworth Sleepiness Scale). Secondary outcomes are other symptoms control, compliance with treatment, metabolic control, quality of life, feasibility, patient and family satisfaction with care, safety, and disease-related costs. At baseline and at 12 months, patients will undergo neurologic, metabolic, and psychosocial

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Clinical Characteristics and Burden of Illness in Pediatric Patients with Narcolepsy

Abstract: Thus, the discrete BOI of pediatric narcolepsy underscores the need for prompt and accurate diagnosis, and appropriate treatment of this disorder.

Cited by 88 publications

References 92 publications

<p>Living with Narcolepsy: Current Management Strategies, Future Prospects, and Overlooked Real-Life Concerns</p>

<p>Living with Narcolepsy: Current Management Strategies, Future Prospects, and Overlooked Real-Life Concerns</p>

Narcolepsy treatment: pharmacological and behavioral strategies in adults and children

Protocols of a diagnostic study and a randomized controlled non-inferiority trial comparing televisits vs standard in-person outpatient visits for narcolepsy diagnosis and care: TElemedicine for NARcolepsy (TENAR)

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