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)

Francesca Ingravallo, Luca Vignatelli, Uberto Pagotto, Stefano Vandi, Monica Moresco, Anastasia Mangiaruga, Claudia Oriolo, Corrado Zenesini, Fabio Pizza, Giuseppe Plazzi, Francesca Ingravallo, Luca Vignatelli, Uberto Pagotto, Stefano Vandi, Monica Moresco, Anastasia Mangiaruga, Claudia Oriolo, Corrado Zenesini, Fabio Pizza, Giuseppe Plazzi

Abstract

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 two-arm, 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 assessments and we will measure primary and secondary outcomes. Primary outcomes will be also measured at 6 months (remotely or in person, according to the arm).

Discussion: TENAR project will assess, for the first time, the feasibility, accuracy, efficacy and safety of Telemedicine procedures applied to the diagnosis and the multidisciplinary care of children and adults with narcolepsy. The study may be a model for the remote management of other rare disorders, offering care access for patients living in areas lacking medical centres with specific expertise.

Trial registration: Number of the Tele-multidisciplinary care study NCT04316286. Registered 20 March 2020.

Keywords: Care; Diagnosis; Management; Narcolepsy; Quality of life; Sleep disorders; Sleepiness; Telemedicine; Televisit.

Conflict of interest statement

The Authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
Flowchart of the randomized controlled trial

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