Development and validation of a disease-specific quality of life scale for adult patients with Fabry disease in Japan

Plain English summary Adults with Fabry disease experience severe challenges, which adversely impact their quality of life (QOL). As it is a rare disease, non-patients lack awareness of the severity of its symptoms and the resultant social difficulties of the patients. Most instruments that measure...

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Bibliographic Details
Main Authors: Yuta Koto, Wakana Yamashita, Yoko Lee, Nozomi Hadano, Chikara Kokubu, Norio Sakai
Format: Article
Language:English
Published: SpringerOpen 2022-11-01
Series:Journal of Patient-Reported Outcomes
Subjects:
Online Access:https://doi.org/10.1186/s41687-022-00525-z
Description
Summary:Plain English summary Adults with Fabry disease experience severe challenges, which adversely impact their quality of life (QOL). As it is a rare disease, non-patients lack awareness of the severity of its symptoms and the resultant social difficulties of the patients. Most instruments that measure QOL are not specific enough to address issues related to Fabry disease. Therefore, in this study, a measurement instrument known as the Adult Fabry QOL (AFQOL) scale was designed and validated. The 39-item scale covers five domains that are congruent with the symptoms of adult Fabry disease. It differs from other QOL scales as it also assesses the impact of work and personal relationships on patients’ QOL and symptoms that progress in adulthood. This study has important implications for healthcare providers who treat adult patients with Fabry disease, enabling them to have a fuller picture of the unique needs of this population.
ISSN:2509-8020