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. 2017 Apr 14;12(1):70.
doi: 10.1186/s13023-017-0621-5.

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

Indraneel Banerjee  1 , Diva De Leon  2 , Mark J Dunne  3   4
Affiliations

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

Indraneel Banerjee et al. Orphanet J Rare Dis. .

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: Congenital hyperinsulinism in Infancy patient; Sirolimus; hypoglycaemia; islet; mTOR.

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Figures

Fig. 1
Fig. 1
Relative expression of mTOR in CHI tissue. The relative expression of mTOR mRNA is no different in focal (n = 5 cases, ‘Lesion’) or diffuse CHI (n = 3 cases, ‘Diffuse’) when compared to age-matched controls (n = 4 cases)
See this image and copyright information in PMC

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