Patient-reported outcomes on empagliflozin treatment in glycogen storage disease type Ib: An international questionnaire study.

ABSTRACT: In patients with glycogen storage disease type Ib (GSD Ib), quality of life is severely hampered by neutropenia and neutropenia-associated symptoms. SGLT2 inhibitors are a new treatment option and have shown improved medical outcomes in more than 120 patients so far. The aim of this international questionnaire study was to assess patient-reported outcomes of this new treatment in GSD Ib patients. Patients and caregivers of pediatric patients were invited to complete a web-based questionnaire. This was designed to evaluate treatment effects of the SGLT2 inhibitor empagliflozin on clinical symptoms and important aspects of daily life including physical performance, sleep, social and work life, traveling, socioeconomic aspects, and quality of life. The questionnaire was completed by 73 respon

SUBMITTER: Grunert SC 

PROVIDER: S-EPMC10159866 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature