ESPE Abstracts

Background: Severe diazoxide unresponsive hyperinsulinism (DUHI) is most often caused by autosomal recessive variants in the KATP channel genes. Because of the limited medical treatments available, many patients are treated with 98% pancreatectomy. This results in a high rate of diabetes by the age of 15 years. Many centers now try to avoid surgery to prevent the inevitable transition to post-surgical diabetes.

Objectives: To report our single center experience of the treatment of DUHI and to determine if there are clinical differences between those treated with surgery versus high intensity medical treatment.

Methods: We performed a retrospective review of children treated for hyperinsulinism at our Congenital Hyperinsulinism Center between January 2014 and March 2023. We compared those with DUHI treated by 98% pancreatectomy versus medical therapy. We evaluated the long term glycemic outcome, length of stay, and …. Data is stored in our IRB approved Hyperinsulinism Registry.

Results: There were 95 patients treated over the study period. 27 (28%) were diazoxide responsive and 68 (72%) DU. Off these 41 (60%) had Focal HI and 27 (40%) had diffuse HI. Of those 27, 8 (30%) were managed medically and 19 (70%) had 98% pancreatectomy. Of the 8 who had no surgery 3 were treated in the Dasiglucagon study, and the remaining 5 were treated with octreotide plus continuous night feeds. Following a lengthy trial of medical therapy 5 of the 8 (63%%) families returned for surgery because medical management was too difficult to continue. Baseline data are shown in Table 1. Of the 19 surgeries, 5 (26%) have diabetes at last follow up, 5 (26%) hypoglycemia and 9 (48%) eat normally for age without hypoglycemia. Age of last follow up is 3 years

Conclusions: Long term medical therapy was attempted in 30% of our DUHI diffuse patients. Only 38% of them were able to continue to manage long term medical therapy and 62% opted to have a 98% pancreatectomy because they were unwilling to continue intensive therapy and felt diabetes was an “easier” path.