Focal congenital hyperinsulinism: spontaneous glycemic improvement before surgery

Congenital hyperinsulinism (CHI) is the most common genetic cause of hypoglycaemia in infants. Severe CHI may result from a focal pancreatic lesion (FoCHI), which can be surgically removed, although postoperative complications may occur, particularly after pancreatic head resections.

To describe the spontaneous glycaemic evolution of FoCHI before surgery and to assess the potential role of conservative management.

We retrospectively reviewed medical records of patients with FoCHI managed at a single referral centre. CHI severity was classified according to the need for continuous gastric tube feeding (TF) to maintain glycaemic control prior to surgery, defining a TF group and a no-tube feeding (NTF) group.

Thirty-nine patients were included: 12 (30.8%) in the NTF group and 27 (69.2%) in the TF group. Patients in the NTF group were diagnosed later (p = 0.022), had less severe initial hypoglycaemia (p = 0.003), and 4/12 did not undergo surgery and were cured of hypoglycaemia before 6 years of age. We developed an exploratory predictive score for early disease evolution, showing a specificity of 97%. Surgery was curative in most patients (96.3% in the TF group, 100% in the NTF group). Long-term complications were uncommon (8.6% vs. 0%) and consisted exclusively of exocrine pancreatic insufficiency, occurring only after pancreatic head surgery (15.8%).

Approximately 30% of infants with FoCHI demonstrated rapid early improvement. While surgery remains the standard curative treatment, a conservative approach may be considered in selected infants with pancreatic head lesions who show early favourable glycaemic evolution, potentially after surgical exploration to assess postoperative risk.