Endocrine and diabetes medicines

Overview & Definition

A child walks in. The endocrine axis that quietly ran their metabolism has failed, and the fix is to give back the missing hormone from the outside. Endocrine prescribing is the deliberate act of choosing the right hormone preparation, the right weight-based dose, the right timing, and the right monitoring — so that the child grows, metabolises and develops as if the gland were still working. [1]

Three medicine families carry almost all of the exam weight, and they are the ones a general paediatrician writes in everyday practice rather than leaving entirely to the specialist. Insulin replaces the hormone the beta-cells can no longer make in type 1 diabetes and is the definitive, switch-off-ketogenesis drug in diabetic ketoacidosis. Levothyroxine replaces the thyroxine the thyroid cannot make in congenital and acquired hypothyroidism, and in the first two years of life it is a neurodevelopmental drug as much as a metabolic one. Recombinant human growth hormone drives linear growth in growth hormone deficiency and a handful of licensed growth-failure indications, and it is a daily, self-injected, years-long commitment that tests adherence and safety surveillance at every visit. [1] [6]

The reason these three dominate is that they are common, they are dangerous when misprescribed, and they sit at the centre of the fellowship written and clinical exams. A child in DKA given an insulin bolus before fluids can die of cerebral oedema; a neonate whose levothyroxine is delayed past the first weeks loses IQ points that never come back; an adolescent on growth hormone with a new headache may have intracranial hypertension. The fellowship candidate is expected to write the dose, defend the regimen, and explain the safety-net — not to defer the whole problem to an endocrinologist. [4] [6]

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1. [1]Cengiz E, Danne T, et al International Society for Pediatric and Adolescent Diabetes Clinical Practice Consensus Guidelines 2024: Insulin and Adjunctive Treatments in Children and Adolescents with Diabetes Horm Res Paediatr, 2024.PMID 39884261
2. [2]de Bock M, Agwu JC, et al International Society for Pediatric and Adolescent Diabetes Clinical Practice Consensus Guidelines 2024: Glycemic Targets Horm Res Paediatr, 2024.PMID 39701064
3. [3]Tauschmann M, Cardona-Hernandez R, et al International Society for Pediatric and Adolescent Diabetes Clinical Practice Consensus Guidelines 2024 Diabetes Technologies: Glucose Monitoring Horm Res Paediatr, 2024.PMID 39884260
4. [4]Wolfsdorf JI, Glaser N, et al ISPAD Clinical Practice Consensus Guidelines 2018: Diabetic ketoacidosis and the hyperglycemic hyperosmolar state Pediatr Diabetes, 2018.PMID 29900641
5. [5]Brown SA, Kovatchev BP, et al Six-Month Randomized, Multicenter Trial of Closed-Loop Control in Type 1 Diabetes N Engl J Med, 2019.PMID 31618560
6. [6]Léger J, Olivieri A, et al European Society for Paediatric Endocrinology consensus guidelines on screening, diagnosis, and management of congenital hypothyroidism J Clin Endocrinol Metab, 2014.PMID 24446653
7. [7]Esposito A, Vigone MC, et al Effect of initial levothyroxine dose on neurodevelopmental and growth outcomes in children with congenital hypothyroidism Front Endocrinol (Lausanne), 2022.PMID 36133316
8. [8]Kanakatti Shankar R, Quigley CA, et al Growth and Growth-Promoting Treatments in Turner Syndrome Am J Med Genet C Semin Med Genet, 2025.PMID 39950365
9. [9]Azova S, Rapaport R, et al Brain injury in children with diabetic ketoacidosis: Review of the literature and a proposed pathophysiologic pathway for the development of cerebral edema Pediatr Diabetes, 2021.PMID 33197066
10. [10]Brown KM, Glaser NS, et al Rehydration Rates and Outcomes in Overweight Children With Diabetic Ketoacidosis Pediatrics, 2023.PMID 37920947
11. [11]Marks BE, Grundman JB, et al Hybrid Closed Loop Systems Improve Glycemic Control and Quality of Life in Historically Minoritized Youth with Diabetes Diabetes Technol Ther, 2024.PMID 38444316
12. [12]Chen SC, Bryce J, et al Development of a Minimum Dataset for the Monitoring of Recombinant Human Growth Hormone Therapy in Children with Growth Hormone Deficiency: A GloBE-Reg Initiative Horm Res Paediatr, 2024.PMID 37703843