Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient | Orphanet Journal of Rare Diseases | Full Text

Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient | Orphanet Journal of Rare Diseases | Full Text





Orphanet Journal of Rare Diseases

Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient

• Indraneel Banerjee,
• Diva De Leon and
• Mark J. DunneEmail author

Orphanet Journal of Rare Diseases201712:70

DOI: 10.1186/s13023-017-0621-5

©  The Author(s). 2017

Received: 25 January 2017

Accepted: 28 March 2017

Published: 14 April 2017

Abstract

We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced by the argument that any benefit for some patients outweighs the potential and later long-term problems that accompany mTOR inhibition in the neonate. We also express the opinion that caution must be taken when repurposing/repositioning therapies in the field of rare disease.

Keywords

Sirolimus Congenital hyperinsulinism in Infancy patient mTOR islet hypoglycaemia