Cargando…

Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy

Congenital hyperinsulinism (CHI) is the most common cause of severe, persistent hypoglycemia in neonates and infants. If the patient does not respond to medical treatment the currently available treatment is subtotal pancreatectomy, but some patients still experience severe hypoglycemia after surger...

Descripción completa

Detalles Bibliográficos
Autores principales: Chen, Qiong, Chen, Yongxing, Wang, Xiaohong, Yang, Haihua, Zhang, Yingxian, Liu, Xiaojing, Yan, Yun, Wei, Haiyan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Galenos Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7947726/
https://www.ncbi.nlm.nih.gov/pubmed/32482020
http://dx.doi.org/10.4274/jcrpe.galenos.2020.2020.0033
_version_ 1783663288479907840
author Chen, Qiong
Chen, Yongxing
Wang, Xiaohong
Yang, Haihua
Zhang, Yingxian
Liu, Xiaojing
Yan, Yun
Wei, Haiyan
author_facet Chen, Qiong
Chen, Yongxing
Wang, Xiaohong
Yang, Haihua
Zhang, Yingxian
Liu, Xiaojing
Yan, Yun
Wei, Haiyan
author_sort Chen, Qiong
collection PubMed
description Congenital hyperinsulinism (CHI) is the most common cause of severe, persistent hypoglycemia in neonates and infants. If the patient does not respond to medical treatment the currently available treatment is subtotal pancreatectomy, but some patients still experience severe hypoglycemia after surgery. Sirolimus, a mammalian target of rapamycin inhibitor has recently 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 subtotal pancreatectomy. The patient had a heterozygous mutation in ABCC8 but was unresponsive to an optimal dose of diazoxide (15 mg/ kg/day) and octreotide (30 μg/kg/day). The patient subsequently had subtotal pancreatectomy but severe and persistent hypoglycemia continued post-operatively. Sirolimus was commenced. There was a remarkable improvement in glycemic control 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. At the time of this report, at an age of three years and eight months, the patient continues to maintain good glycemic control. This report suggests that sirolimus may be an effective treatment option in patients with CHI resistant to established medical therapy or failure of subtotal pancreatectomy. However, the long-term safety requires study in larger groups of very young patients.
format Online
Article
Text
id pubmed-7947726
institution National Center for Biotechnology Information
language English
publishDate 2021
publisher Galenos Publishing
record_format MEDLINE/PubMed
spelling pubmed-79477262021-03-23 Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy Chen, Qiong Chen, Yongxing Wang, Xiaohong Yang, Haihua Zhang, Yingxian Liu, Xiaojing Yan, Yun Wei, Haiyan J Clin Res Pediatr Endocrinol Case Report Congenital hyperinsulinism (CHI) is the most common cause of severe, persistent hypoglycemia in neonates and infants. If the patient does not respond to medical treatment the currently available treatment is subtotal pancreatectomy, but some patients still experience severe hypoglycemia after surgery. Sirolimus, a mammalian target of rapamycin inhibitor has recently 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 subtotal pancreatectomy. The patient had a heterozygous mutation in ABCC8 but was unresponsive to an optimal dose of diazoxide (15 mg/ kg/day) and octreotide (30 μg/kg/day). The patient subsequently had subtotal pancreatectomy but severe and persistent hypoglycemia continued post-operatively. Sirolimus was commenced. There was a remarkable improvement in glycemic control 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. At the time of this report, at an age of three years and eight months, the patient continues to maintain good glycemic control. This report suggests that sirolimus may be an effective treatment option in patients with CHI resistant to established medical therapy or failure of subtotal pancreatectomy. However, the long-term safety requires study in larger groups of very young patients. Galenos Publishing 2021-03 2021-02-26 /pmc/articles/PMC7947726/ /pubmed/32482020 http://dx.doi.org/10.4274/jcrpe.galenos.2020.2020.0033 Text en ©Copyright 2021 by Turkish Pediatric Endocrinology and Diabetes Society | The Journal of Clinical Research in Pediatric Endocrinology published by Galenos Publishing House. http://creativecommons.org/licenses/by/2.5/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Case Report
Chen, Qiong
Chen, Yongxing
Wang, Xiaohong
Yang, Haihua
Zhang, Yingxian
Liu, Xiaojing
Yan, Yun
Wei, Haiyan
Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title_full Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title_fullStr Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title_full_unstemmed Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title_short Sirolimus Therapy and Follow-up in a Patient with Severe Congenital Hyperinsulinism Following Subtotal Pancreatectomy
title_sort sirolimus therapy and follow-up in a patient with severe congenital hyperinsulinism following subtotal pancreatectomy
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7947726/
https://www.ncbi.nlm.nih.gov/pubmed/32482020
http://dx.doi.org/10.4274/jcrpe.galenos.2020.2020.0033
work_keys_str_mv AT chenqiong sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT chenyongxing sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT wangxiaohong sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT yanghaihua sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT zhangyingxian sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT liuxiaojing sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT yanyun sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy
AT weihaiyan sirolimustherapyandfollowupinapatientwithseverecongenitalhyperinsulinismfollowingsubtotalpancreatectomy