Sirolimus Therapy in Infants with Severe Hyperinsulinemic Hypoglycemia
Senthil Senniappan, M.D., Sanda Alexandrescu, M.D., Nina Tatevian, M.D., Pratik Shah, M.D., Ved Arya, M.D., Sarah Flanagan, Ph.D., Sian Ellard, Ph.D., Dyanne Rampling, F.I.B.M.S., Michael Ashworth, M.D., Robert E. Brown, M.D., and Khalid Hussain, M.D.
N Engl J Med 2014; 370:1131-1137March 20, 2014DOI: 10.1056/NEJMoa1310967
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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.