ESPE2023 Poster Category 1 Fetal, Neonatal Endocrinology and Metabolism (34 abstracts)
Low-Dose Diazoxide Therapy in Hyperinsulinaemic Hypoglycaemia
Amy Yi-Lin Ng 1,2 , Pankaj Agarwal 3 , Roopa Vijayan 3 , Ved B Arya 3,4 , Ritika R Kapoor 3,4 & Pratik Shah 2,5
1Medical Student, Barts and The London School of Medicine & Dentistry, London, United Kingdom. 2Department of Paediatric Endocrinology and Diabetes, The Royal London Children's Hospital, Barts Health NHS Trust, London, United Kingdom. 3Department of Paediatric Endocrinology, Variety Children's Hospital, King's College Hospital NHS Foundation Trust, London, United Kingdom. 4King’s College London, Faculty of Medicine and Life Science, London, United Kingdom. 5Centre for Endocrinology, William Harvey Research Institute, Queen Mary University of London, London, United Kingdom
Background: Diazoxide therapy is used as first line treatment in hyperinsulinaemic hypoglycaemia (HH). Apart from a single study reporting efficacy of low dose diazoxide in small for gestational age (SGA) infants, diazoxide has been reported to be used in doses of 5-20 mg/kg/day [1].
Objective: To report the outcomes of infants with HH responsive to low dose diazoxide (≤5mg/kg/day).
Methods: Retrospective analysis of 34 patients with biochemically confirmed HH that were treated with low-dose diazoxide at two Tertiary Children Hospitals in London from April 2020 to March 2023. Patient characteristics and treatment details were collected from electronic patient records. SGA was defined as birth weight <10th centile.
Results: Of the 34 patients, 15 were SGA. The patient characteristics and details of diazoxide treatment are summarised in Table 1. All infants underwent an age appropriate controlled fast successfully prior to discharge from the hospital. During follow-up, 6 (17.6%) babies required minimal increase in the dose of diazoxide for borderline blood glucose concentrations, with the total daily dosage still remaining ≤5mg/kg/day. 5 (14.7%) patients were identified to have minimal fluid retention during follow-up which resolved on increasing the dose of diuretics. 2 patients had a genetic confirmation of HNF4A mutation, done in view of the strong family history of diabetes. No significant neurodevelopmental concerns have been identified on follow-up so far.
| SGA (n=15) | Non-SGA (n=19) | |||
| Preterm | Term | Preterm | Term | |
| Median birth weight (g) | 1810 | 2385 | 2680 | 3150 |
| Median birth weight standard deviation score | -3.20 | -2.02 | -0.0515 | -0.0410 |
| Gestational age range (weeks) | 33-36+6 | 37-41 | 24-36+6 | 38-41+6 |
| Median dose of diazoxide on discharge from hospital (mg/kg/day) | 3.00 | 2.74 | 2.48 | 3.00 |
| Median starting age of diazoxide (days) | 17 | 12 | 19 | 15 |
| Median age of stopping diazoxide (months) | 5.5 | 4 | 4 | 4 |
Conclusion: Low-dose diazoxide is effective treatment for babies with HH, independent of birth weight. Certain genetic forms of HH may also be suitable for treatment with low dose diazoxide. Hence, lower doses of diazoxide should be considered in infants with HH prior to using traditionally published doses (>5 mg/kg/day). Although generally well-tolerated, fluid retention can develop in a minority of patients, confirming a need for regular follow-ups and vigilance even at lower doses.
References: 1. Chandran S et al. Safety and efficacy of low-dose diazoxide in small-for-gestational-age infants with hyperinsulinaemic hypoglycaemia. Arch Dis Child Fetal Neonatal Ed. 2022 Jul;107(4):359-363. doi:10.1136/archdischild-2021-322845.
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