Sirolimus therapy and follow-up in a patient with severe congenital hyperinsulinism following subtotal pancreatectomy.

Abstract:

:Congenital hyperinsulinism (CHI) is the most common cause of severe, persistent hypoglycemia in neonates and infants. Current available treatment is subtotal pancreatectomy if the patient does not respond to medical treatment but some of the patients still experience severe hypoglycemia after the surgery. Sirolimus, a mammalian target of rapamycin(mTOR) inhibitor recently has been reported to be effective in the treatment of insulinoma and CHI patients. Here we report a patient with CHI who had prolonged hypoglycemia after the subtotal pancreatectomy. The patient with a heterozygous mutation in ABCC8 was unresponsive to optimal dose of diazoxide (15 mg/kg/day) and octreotide (30µg/kg/day). The patient subsequently had subtotal pancreatectomy; however, the patient continued to have severe and persistent hypoglycemia. Sirolimus was commenced. Glycemic control had remarkable improvement without major adverse events, although he required a small dose of octreotide to maintain euglycemia. Sirolimus therapy was discontinued when the patient was 15 months old. The patient had been having a good glycemic control at the time of this report when he was three years and 8 months old. This report suggests that sirolimus may be an effective treatment option in patients with CHI resistant to traditional medical therapy or failure of subtotal pancreatectomy. However, the long-term safety needs to be studied in larger group of very young patients.

authors

Chen Q,Chen Y,Wang X,Yang H,Zhang Y,Liu X,Yan Y,Wei H

doi

10.4274/jcrpe.galenos.2020.2020.0033

subject

Has Abstract

pub_date

2020-06-02 00:00:00

eissn

1308-5727

issn

1308-5735

pub_type

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