Objectives Diazoxide (DZX) is the drug of choice for treating hyperinsulinaemic hypoglycaemia (HH), and it has potentially serious adverse effects. We studied the safety and efficacy of low-dose DZX in small-for-gestational-age (SGA) infants with HH.
Design An observational cohort study from 1 September 2014 to 31 September 2020.
Setting A tertiary Women’s and Children’s Hospital in Singapore.
Patients All SGA infants with HH.
Intervention Diazoxide, at 3–5 mg/kg/day.
Main outcome measures Short-term outcomes; adverse drug events and fasting studies to determine ‘safe to go home’ and ‘resolution’ of HH.
Results Among 71 836 live births, 11 493 (16%) were SGA. Fifty-six (0.5%) SGA infants with HH were identified, of which 27 (47%) with a mean gestational age of 36.4±2 weeks and birth weight of 1942±356 g required DZX treatment. Diazoxide was initiated at 3 mg/kg/day at a median age of 10 days. The mean effective dose was 4.6±2.2 mg/kg/day, with 24/27 (89%) receiving 3–5 mg/kg/day. Generalised hypertrichosis occurred in 2 (7.4%) and fluid retention in 1 (3.7%) infant. A fasting study was performed before home while on DZX in 26/27 (96%) cases. Diazoxide was discontinued at a median age of 63 days (9–198 days), and resolution of HH was confirmed in 26/27 (96%) infants on passing a fasting study.
Conclusion Our study demonstrates that low-dose DZX effectively treats SGA infants with HH as measured by fasting studies. Although the safety profile was excellent, minimal adverse events were still observed with DZX, even at low doses.
Data availability statement
Data are available on reasonable request. The data that support the findings of this study are available from the corresponding author on reasonable request.
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SC and FY contributed equally.
Contributors SC and FY conceptualised and designed the study, wrote the manuscript and performed the final edits. Both authors supervised the treatment of patients and coordinated the hyperinsulinism service. PRR drafted the initial manuscript and inserted the references. CMC and VSR reviewed and revised the manuscript critically for important intellectual content. SES is the biostatistician who is responsible for the data analysis and interpretation. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.