Work

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

Year: 2017

Type: article

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.

Source: Orphanet Journal of Rare Diseases

DOI: 10.1186/s13023-017-0621-5

Authors: Indraneel Banerjee, Diva De Leon, Mark J. Dunne

Institutions: University of Manchester, Children's Hospital of Philadelphia

Language: English

Topic: Pancreatic function and diabetes

Subfield: Surgery

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,003