西罗莫司治疗严重高胰岛素血症性低血糖症婴儿。
Sirolimus therapy in infants with severe hyperinsulinemic hypoglycemia.
机构信息
From the Developmental Endocrinology Research Group, Clinical and Molecular Genetics Unit, Institute of Child Health, University College London (S.S., P.S., V.A., K.H.), and the Departments of Paediatric Endocrinology (S.S., P.S., V.A., K.H.) and Histopathology (D.P., M.A.), Great Ormond Street Hospital for Children, London, and the Institute of Biomedical and Clinical Science, University of Exeter Medical School, Exeter (S.F., S.E.) - all in the United Kingdom; the Department of Pathology, University of California, San Francisco, San Francisco (S.A.); and the Department of Pathology and Laboratory Medicine, University of Texas Medical School at Houston, Houston (N.T., R.E.B.).
出版信息
N Engl J Med. 2014 Mar 20;370(12):1131-7. doi: 10.1056/NEJMoa1310967.
Hyperinsulinemic hypoglycemia is the most common cause of severe, persistent neonatal hypoglycemia. The treatment of hyperinsulinemic hypoglycemia that is unresponsive to diazoxide is subtotal pancreatectomy. We examined the effectiveness of the mammalian target of rapamycin (mTOR) inhibitor sirolimus in four infants with severe hyperinsulinemic hypoglycemia that had been unresponsive to maximal doses of diazoxide (20 mg per kilogram of body weight per day) and octreotide (35 μg per kilogram per day). All the patients had a clear glycemic response to sirolimus, although one patient required a small dose of octreotide to maintain normoglycemia. There were no major adverse events during 1 year of follow-up.
高胰岛素血症性低血糖是严重、持续性新生儿低血糖的最常见原因。对于对二氮嗪无反应的高胰岛素血症性低血糖的治疗是胰腺次全切除术。我们研究了雷帕霉素（mTOR）抑制剂西罗莫司在 4 例对最大剂量二氮嗪（每天 20 毫克/千克体重）和奥曲肽（每天 35 微克/千克）无反应的严重高胰岛素血症性低血糖婴儿中的有效性。所有患者对西罗莫司均有明确的血糖反应，尽管有 1 例患者需要小剂量奥曲肽维持血糖正常。在 1 年的随访中没有发生重大不良事件。
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