Endocrine Abstracts

Background: Transient Hyperinsulinism (HI) often resolves within the first year of life, but many affected infants require temporary treatment with diazoxide, a medication associated with side effects including pulmonary hypertension, fluid retention, and hypertrichosis. Historically, discontinuation of diazoxide required hospital admission for glucose monitoring and fasting tolerance testing, typically scheduled between 6–12 months of age. During the COVID-19 pandemic, restrictions delayed inpatient admissions, potentially prolonging unnecessary exposure to diazoxide therapy.

Objective: To reduce delays in re-evaluating the need for diazoxide therapy and unwarranted exposure to associated risks, we developed a new CNS-led pathway allowing parents/carers to safely stop diazoxide at home under structured guidance and support from the HI specialist nursing team.

Methods: The pathway included clear education for parents/carers, regular telephone check-ins, and ongoing home blood glucose monitoring. Once off diazoxide, infants were admitted for a single overnight fasting tolerance test, rather than the previous 48-hour inpatient stay with a 24-hour glucose profile. A service satisfaction survey was conducted in 2023 to evaluate family experience with the pathway.

Results: Implementation of the pathway led to a significant reduction in time on diazoxide therapy, with the average age of discontinuation reduced to 3 months. Hospital admission duration was halved, reducing burden on families and improving inpatient efficiency and financial benefit. Feedback from 30 families showed 100% recalled discussions about the pathway, with 72% strongly agreeing and 24% agreeing they felt confident stopping diazoxide at home. Parents appreciated shorter admissions, fewer medication side effects, and improved feeding flexibility.

Conclusion: This CNS-led pathway enabled earlier discontinuation of diazoxide, reducing exposure to side effects and improving quality of life for infants and families. It also led to cost savings through reduced bed days and medication use. Positive parental feedback supports continued use of the pathway.