Cao Bingyan, Di Wu, Su Chang, Chen Jiajia, Liang Xuejun, Liu Min, Li Wenjing, Li Xiaoqiao, Gong Chunxiu
Department of Endocrinology, Genetics and Metabolism Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Pediatr Investig. 2020 Mar 17;4(1):29-36. doi: 10.1002/ped4.12175. eCollection 2020 Mar.
Octreotide is an off-label medicine for congenital hyperinsulinism (CHI), but is currently widely used for treatment of patients with CHI. Thus far, variable efficacy and adverse effects have been reported for octreotide.
The present study evaluated the efficacy and safety of a subcutaneous octreotide injection for treatment of diazoxide-unresponsive CHI in China.
This study was a retrospective review of children with diazoxide-unresponsive CHI who were treated with a subcutaneous octreotide injection. The efficacy and side effects of the treatment were assessed.
Twenty-five Chinese children (15 boys) were involved in the study. Their median age at diagnosis was 8 weeks (range, 1-24 weeks) and median age at the final follow-up was 1.8 years (range, 0.3-3.3 years). Octreotide therapy effectively increased blood glucose levels in all patients. The intravenous glucose infusion rate was reduced in all patients. Twenty-one patients gradually discontinued the intravenous glucose infusion while receiving octreotide combined with frequent carbohydrate/glucose-rich feeding. Among patients with a monoallelic ATP-sensitive potassium (KATP) channel mutation, 50.0% showed gradual remission during follow up, indicating that the octreotide treatment may be a feasible alternative to surgery, especially for patients with monoallelic KATP-channel mutations. Transient elevation of liver enzymes occurred in 20.0% of patients, while asymptomatic gallbladder pathology occurred in one patient. The growth rates of these patients were normal (height standard deviation score was 0.3 ± 1.5 at the final follow-up).
Octreotide was a well-tolerated, effective therapy for most children with diazoxide-unresponsive CHI.
奥曲肽是一种用于先天性高胰岛素血症（CHI）的非适应证用药，但目前广泛用于CHI患者的治疗。迄今为止，关于奥曲肽的疗效和不良反应报道不一。
本研究评估皮下注射奥曲肽治疗中国二氮嗪无反应性CHI的疗效和安全性。
本研究对接受皮下注射奥曲肽治疗的二氮嗪无反应性CHI患儿进行回顾性分析，评估治疗的疗效和副作用。
25名中国儿童（15名男孩）参与了本研究。他们诊断时的中位年龄为8周（范围1 - 24周），末次随访时的中位年龄为1.8岁（范围0.3 - 3.3岁）。奥曲肽治疗有效提高了所有患者的血糖水平，所有患者的静脉葡萄糖输注速率均降低。21名患者在接受奥曲肽联合频繁富含碳水化合物/葡萄糖喂养时逐渐停止了静脉葡萄糖输注。在单等位基因ATP敏感性钾（KATP）通道突变的患者中，50.0%在随访期间逐渐缓解，这表明奥曲肽治疗可能是手术的可行替代方案，特别是对于单等位基因KATP通道突变的患者。20.0%的患者出现肝酶短暂升高，1名患者出现无症状胆囊病变。这些患者的生长速率正常（末次随访时身高标准差评分为0.3±1.5）。
对于大多数二氮嗪无反应性CHI儿童，奥曲肽是一种耐受性良好且有效的治疗方法。
