Supplementary Material for: Long-Term Lanreotide Treatment in Six Patients with Congenital Hyperinsulinism

<b><i>Background:</i></b> Medical treatment is a substantial therapeutic measure to achieve glycemic control and prevent hypoglycemic brain damage without surgery in patients with congenital hyperinsulinism (CHI). However, only few drugs are available and even fewer are approved as a medical therapy to maintain normal blood glucose levels. The established therapies are demanding for caregivers and complicated by different side effects such as gastrointestinal symptoms, hypertrichosis, and obesity. Therefore, it is important to develop new strategies to improve blood glucose control. <b><i>Methods:</i></b> We report the use of the very-long-acting somatostatin analogue lanreotide autogel in 6 patients with CHI over a mean duration of 40.8 months. Blood glucose levels before and after the start and dosage titration of lanreotide in these patients are compared. <b><i>Results:</i></b> In 3 of 6 patients, switching to lanreotide raised mean blood glucose levels and reduced individually as well as overall the risk for hypoglycemic episodes (odds ratio 0.38) significantly. <b><i>Conclusion:</i></b> Lanreotide autogel can be used as an alternative pharmacological treatment and may be beneficial in conservatively treated patients with CHI.

<b><i>背景:</i></b> 先天性高胰岛素血症（congenital hyperinsulinism, CHI）患者无需接受手术即可实现血糖控制、预防低血糖性脑损伤，药物治疗是此类患者的重要治疗手段。但目前临床可用药物寥寥无几，获批用于维持正常血糖水平的治疗药物更是屈指可数。现有标准治疗方案不仅对护理人员要求较高，还易引发多种不良反应，如胃肠道症状、多毛症及肥胖。因此，开发改善血糖控制的新策略具有重要临床意义。<b><i>方法:</i></b> 本研究纳入6例先天性高胰岛素血症患者，为其应用超长效生长抑素类似物兰瑞肽自释微球（lanreotide autogel）进行治疗，平均随访时长为40.8个月。研究对比了患者在启动兰瑞肽治疗及调整给药剂量前后的血糖水平。<b><i>结果:</i></b> 6例患者中的3例在换用兰瑞肽治疗后，平均血糖水平显著提升，个体及总体低血糖发作风险均明显降低（优势比（odds ratio）为0.38）。<b><i>结论:</i></b> 兰瑞肽自释微球可作为先天性高胰岛素血症保守治疗的替代药物方案，有望为此类患者带来临床获益。