@article {8059,
	title = {Sirolimus Therapy in Congenital Hyperinsulinism: A Successful Experience Beyond Infancy.},
	journal = {Pediatrics},
	volume = {136},
	year = {2015},
	month = {2015 Nov},
	pages = {e1373-6},
	abstract = {<p>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.</p>},
	issn = {1098-4275},
	doi = {10.1542/peds.2015-1132},
	author = {Minute, Marta and Patti, Giuseppa and Tornese, Gianluca and Faleschini, Elena and Zuiani, Chiara and Ventura, Alessandro}
}
