探讨奥曲肽联合西罗莫司对二氮嗪治疗无效的先天性高胰岛素血症(CHI)患儿的治疗效果及安全性。

选择2021年8月至2024年8月苏州大学附属儿童医院收治的二氮嗪治疗无效的122例CHI患儿为研究对象。采用回顾性分析方法，根据治疗方法将其分为研究组(n＝40，采用奥曲肽联合西罗莫司治疗)，对照组1(n＝41，采用奥曲肽治疗)及对照组2(n＝41，采用西罗莫司治疗)。采用χ²检验，对3组CHI患儿治疗有效率及其他不良反应和合并症总发生率进行总体及两两比较；采用单因素方差分析及LSD-t检验，以及配对t检验，对治疗前及治疗3个月后患儿血清空腹血糖(FBG)、空腹胰岛素(FINS)、生长激素(GH)、血清肌酐(Scr)、血尿素氮(BUN)、丙氨酸转氨酶(ALT)、天冬氨酸转氨酶(AST)水平分别进行3组总体及两两比较，以及组内比较。本研究经苏州大学附属儿童医院伦理委员会批准(审批文号：2025CS163)，所有患儿监护人签署临床研究知情同意书。

①3组CHI患儿中，男性占比、入院日龄等一般临床资料比较，差异均无统计学意义(P>0.05)。②研究组及对照组1、2患儿的治疗有效率分别为92.5%(37/40)、68.3%(28/41)及75.6%(31/41)，研究组CHI患儿治疗有效率分别高于对照组1、2，并且差异均有统计学意义(χ²＝7.485、P＝0.006，χ²＝4.287、P＝0.038)；对照组1与对照组2治疗有效率比较，差异无统计学意义(χ²＝0.544、P＝0.461)。③治疗3个月后组间比较结果显示，研究组CHI患儿血清FBG水平分别高于对照组1、2，血清FINS水平分别低于对照组1、2，并且差异均有统计学意义(P<0.05)；组内比较结果显示，各组CHI患儿治疗3个月后血清FBG水平均高于治疗前，血清FINS水平则均低于治疗前，并且差异均有统计学意义(P<0.05)。④3组CHI患儿治疗前、治疗3个月后血清GH、Scr、BUN、ALT、AST水平组间及组内比较，以及其他不良反应和合并症总发生率组间比较，差异均无统计学意义(P>0.05)。

奥曲肽联合西罗莫司对二氮嗪治疗无效CHI患儿，可有效提高治疗效果，促进血糖和胰岛素水平恢复。该方案用药安全，短期内对患儿GH水平及肝、肾功能无不良影响。

To investigate the therapeutic efficacy and safety of octreotide combined with sirolimus in children with congenital hyperinsulinemia (CHI) unresponsive to diazoxide treatment.

From August 2021 to August 2024, a total of 122 children with CHI unresponsive to diazoxide treatment who were hospitalized in the Children′s Hospital of Soochow University were selected as the study subjects. Using a retrospective analysis method, they were divided into study group (n=40, treated with octreotide combined with sirolimus), control group 1 (n=41, treated with octreotide), and control group 2 (n=41, treated with sirolimus) according to treatment methods. Chi-square test was used for overall and pairwise comparisons of the treatment effective rate, and the total incidence rate of other adverse reactions and complications among the three groups. One-way ANOVA and LSD-t test, and paired t-test were used for overall and pairwise comparisons among the three groups, as well as for intragroup comparisons, regarding the levels of children′s serum fasting blood glucose (FBG), fasting insulin (FINS), growth hormone (GH), serum creatinine (Scr), blood urea nitrogen (BUN), alanine aminotransferase (ALT), and aspartate aminotransferase (AST) before treatment and after 3 months of treatment. This study was approved by the Ethics Committee of the Children′s Hospital of Soochow University (Approval No. 2025CS163). Informed consent was obtained, and the clinical research informed consent forms were signed by the guardians of all children.

① There were no significant differences among three groups of CHI children in general clinical data (P>0.05), such as the proportion of male children and age of admission, etc.. ② The treatment effective rates in study group, control group 1, and control group 2 were 92.5% (37/40), 68.3% (28/41), and 75.6% (31/41), respectively. The treatment effective rate in study group was higher than that in control group 1 and control group 2, respectively, and the differences were statistically significant (χ²=7.485, P=0.006; χ²=4.287, P=0.038). There was no significant difference between control group 1 and control group 2 in treatment effective rate (χ²=0.544, P=0.461). ③ The intergroup comparison results after 3 months of treatment showed that the serum FBG level in study group was higher than that in control group 1 and control group 2, respectively, and the serum FINS level was lower than that in control group 1 and control group 2, respectively, and the differences were statistically significant (P<0.05). Intragroup comparisons results showed that the serum FBG levels in each group were higher than that before treatment, respectively, and the serum FINS levels were lower than that before treatment, respectively, and the differences were statistically significant (P<0.05). ④ No significant differences were observed in intergroup or intragroup comparisons of serum GH, Scr, BUN, ALT, and AST levels before and after 3 months of treatment among three groups of CHI children (P>0.05), as well as intergroup comparison of the total incidence rates of other adverse reactions and complications (P>0.05).

Octreotide combined with sirolimus can effectively improve the therapeutic efficacy and promote the recovery of blood glucose and insulin levels in children with CHI unresponsive to diazoxide treatment. This regimen is safe for use and has no adverse effects on the children′s GH level, and liver or kidney function in the short term.