Safety and efficacy of low-dose diazoxide in small-for-gestational-age infants with hyperinsulinaemic hypoglycaemia

Suresh Chandran1,2,3, Pravin R R4, Chua Mei Chien5,2,3

  • 1Department of Neonatology, KK Women's and Children's Hospital, Singapore profschandran2019@gmail.com fabian.yap.k.p@singhealth.com.sg.

Insights

Low-dose diazoxide (DZX) effectively treats hyperinsulinaemic hypoglycaemia (HH) in small-for-gestational-age (SGA) infants. The treatment showed an excellent safety profile with minimal adverse events, confirming its efficacy in this population.

Area of Science:

  • Neonatal Medicine
  • Pediatric Endocrinology
  • Pharmacology

Background:

  • Hyperinsulinaemic hypoglycaemia (HH) in small-for-gestational-age (SGA) infants can lead to serious adverse effects.
  • Diazoxide (DZX) is a primary treatment for HH but carries risks of significant side effects.
  • The safety and efficacy of low-dose DZX in SGA infants with HH require further investigation.

Purpose of the Study:

  • To evaluate the safety and efficacy of low-dose diazoxide (DZX) in treating hyperinsulinaemic hypoglycaemia (HH).
  • To assess short-term outcomes, including adverse drug events and resolution of HH, in small-for-gestational-age (SGA) infants.

Main Methods:

  • An observational cohort study conducted between September 2014 and September 2020 at a tertiary women's and children's hospital.
  • Included SGA infants diagnosed with HH requiring treatment with diazoxide at 3-5 mg/kg/day.
  • Evaluated adverse events and utilized fasting studies to determine treatment success and readiness for discharge.

Main Results:

  • Out of 71,836 live births, 16% were SGA, and 0.5% of SGA infants (56 total) had HH.
  • Twenty-seven SGA infants with HH were treated with diazoxide (mean dose 4.6±2.2 mg/kg/day), with 89% receiving 3-5 mg/kg/day.
  • Minimal adverse events were observed: generalized hypertrichosis in 7.4% and fluid retention in 3.7%. Resolution of HH was confirmed in 96% of infants via fasting studies.

Conclusions:

  • Low-dose diazoxide is an effective treatment for hyperinsulinaemic hypoglycaemia in small-for-gestational-age infants.
  • The observed safety profile of low-dose DZX in this cohort was excellent, with a low incidence of adverse events.
  • Fasting studies are a reliable method to confirm HH resolution and determine when infants are safe for discharge.
Abstract

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