Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective.

ABSTRACT:

Background

Late-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients' daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform the development of new patient-reported outcome (PRO) tools.

Methods

A preliminary conceptual model was constructed following a literature review and revised through interviews with expert clinicians to identify important and relevant concepts regarding symptoms and impacts of LOPD. This preliminary model informed the development of a qualitative patient interview guid

SUBMITTER: Hamed A 

PROVIDER: S-EPMC8513325 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature