Real-world experience with the use of diazoxide among people living with congenital hyperinsulinism and their caregivers.

ABSTRACT:

Introduction

Congenital hyperinsulinism (HI) is a rare disease that causes severe hypoglycemia. Diazoxide is the first-line treatment; however, many individuals using diazoxide continue to experience hypoglycemia. Diazoxide is associated with side effects that impact life and well-being.

Methods

The study utilized a mixed-methods approach combining structured, survey-based cross-sectional quantitative data from the HI Global Registry (HIGR) (n=165, 89% were caregivers), of whom 75% reported current diazoxide use, with qualitative interviews with caregivers (n=12) and individuals with HI (n=6). This is the first mixed-methods study to focus on the experience of diazoxide treatment as reported by the individual taking the medicine and/or their caregiver.

Results

Of HIGR

SUBMITTER: Pasquini TLS 

PROVIDER: S-EPMC12410177 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature