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N Engl J Med. 2014 Mar 20;370(12):1131-7. doi: 10.1056/NEJMoa1310967.

Sirolimus therapy in infants with severe hyperinsulinemic hypoglycemia.

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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.).


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.

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