Backgrounds: The treatment of diazoxide-unresponsive congenital hyperinsulinism (CHI) is a big challenge in clinical practice. Octreotide is an off-lable medicine for CHI but widely used nowadays. However, the efficacy and adverse effects have been reported varied in centers.
Objective: To evaluate the efficacy and safety of the subcutaneous octreotide injection for diazoxide-unresponsive CHI in China.
Subjects and methods: Diazoxide-unresponsive CHI children treated with subcutaneous octreotide injection at an adjusted dosage of up to 50 μg/kg per day were involved in the study. Octreotide is ineffective when blood glucose (BG) <2.8 mmol/l or is completely effective if BG >3.3 mmol/l over 48 h by every 2 h check after wean-off iv dextrose. BG between the above means partial effectiveness. We also test CHI genes by next generation sequencing.
Results: Twenty-five Chinese (15 males) children were enrolled in the study. Their median onset age was 1 day (range 1150 days), the median diagnosis age was 7.5 weeks (range 124 wk), the mean age of last visit was (1.6±0.9) (maximum 3.3) years. The effective median dose of octreotide required was 10.0 (range 1.220.0) μg/kg per day and the BG stabilized at (7.3±2.0) days, the mean duration was (8.9±6.3) months (range 1.525 mo). Eighty-eight percent (22/25) were confirmed with ATP sensitive potassium channel gene mutation (19ABCC8, 3KCNJ11). The octreotide was completely effective, partly effective in 12, 9 patients respectively and ineffective in 4 (16%). The effectiveness (included complete and partial effectiveness) of octreotide was not different between gene-positive and -negative group. The dose of octreotide was not different between monoalliec and biallelic ATP sensitive potassium channel mutation. Transient elevation of liver enzymes occurred in 20% patients, asymptomatic gallbladder pathology occurred in 1 patient. The growth charts of this cohort patients were in normal range (mean height SDS was 0.3±1.5 at the last follow-up).
Conclusions: The octreotide was well tolerated, effective therapy for diazoxide unresponsive CHI cases. It could be a choice for diazoxide-unresponsive patients.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology