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首页> 外文期刊>Pediatrics: Official Publication of the American Academy of Pediatrics >Sirolimus Therapy in Congenital Hyperinsulinism: A Successful Experience Beyond Infancy
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Sirolimus Therapy in Congenital Hyperinsulinism: A Successful Experience Beyond Infancy

机译：先天性高胰岛素血症的西罗莫司疗法：婴儿期以外的成功经验

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摘要

Congenital hyperinsulinism (CHI) due to diffuse involvement of the pancreas is a challenging and severe illness in children. Its treatment is based on chronic therapy with diazoxide and/or octreotide, followed by partial pancreatectomy, which is often not resolutive. Sirolimus, a mammalian target of rapamycin inhibitor, was reported to be effective in treating CHI in infants. We report here the case of an 8-year-old boy affected by a severe form of CHI due to a biallelic heterozygous ABCC8 mutation who responded to sirolimus with a dramatic improvement in his glucose blood level regulation and quality of life, with no serious adverse events after 6 months of follow-up. To the best of our knowledge, this is the first report of a successful intervention in an older child. It provides a promising basis for further studies comparing sirolimus with other treatments, particularly in older children.
机译：由于胰腺的广泛累及引起的先天性高胰岛素血症（CHI）是儿童的一项艰巨而严重的疾病。它的治疗基于以二氮嗪和/或奥曲肽进行的慢性治疗，然后进行部分胰腺切除术，而后者通常无法解决。据报道，西拉莫司是雷帕霉素抑制剂的哺乳动物靶标，可有效治疗婴儿的CHI。我们在这里报告了一个8岁男孩因双等位基因杂合ABCC8突变而受到严重CHI感染的病例，他对西罗莫司有反应，他的血糖水平调节和生活质量得到了显着改善，而没有严重不良反应随访6个月后发生的事件。据我们所知，这是首次成功干预年龄较大的孩子的报道。它为西罗莫司与其他治疗方法（特别是大龄儿童）之间的比较提供了有希望的基础。

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