Patient-reported outcome measures in central disorders of hypersomnolence: consensus of a sleep consortium/RARE-X expert working group

TL;DR

Experts and patients collaborate to identify key outcome measures for studying central disorders of hypersomnolence, aiming to improve diagnosis and treatment.

Contribution

A community-led working group defines patient- and caregiver-reported outcome measures to standardize CDoH research.

Findings

An expert working group identified essential clinical outcome measures for evaluating CDoH symptoms and impacts.

The measures were implemented in a direct-to-patient online study to capture patient and caregiver experiences.

The initiative aims to support standardization of assessments in CDoH research, particularly for primary disorders.

Abstract

Central disorders of hypersomnolence (CDoH), including the primary hypersomnolence disorders of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and Kleine-Levin syndrome (KLS), as well as secondary hypersomnolence disorders, represent an underdiagnosed and under-treated population. Continuing advancements in understanding and treating CDoH rely on an understanding of the patient and caregiver experience. To address this need, a community-led, patient-owned online research study was launched by the nonprofit organizations Sleep Consortium and Global Genes, using the RARE-X research platform. An expert working group of stakeholders with expertise in hypersomnolence disorders, including clinicians, therapy developers, and patient advocates, was convened to identify key patient- and caregiver-reported clinical outcome measures essential for evaluating CDoH symptoms and impacts. These clinical outcome measures have been implemented as part of an online direct-to-patient study. The measures chosen by the Sleep Consortium Expert Working Group are presented here with the hope of supporting the standardization of clinical outcome assessments being used in CDoH research, especially for primary hypersomnolence disorders.

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