Treatment of Glycogen Storage Disease Type I with Hepatocellular Adenoma
Hepatocellular adenoma is a recognised complication in patients with glycogen storage disease type I (GSD-I). Its presence significantly influences the clinical management strategy and may require escalation beyond conservative measures.
Clinical Scenario
This protocol addresses GSD-I patients in whom hepatocellular adenoma has developed. Initial management of liver adenomas in this population is conservative; however, specific clinical circumstances necessitate a more definitive approach.
Treatment Approach
For patients who have not responded adequately to conservative measures, a definitive surgical intervention targeting the liver may be considered — subject to specific eligibility criteria outlined in the full protocol.
Treatment Goals
Successful treatment aims for resolution of metabolic derangement, including correction of hypoglycemia, lactic acidosis, hyperuricemia, and hyperlipidemia.
References
DOI: 10.1038/gim.2014.128
Initially, the management of liver adenomas in the GSD I population should be conservative (Box 3).
Transplantation should be reserved for patients who have not had success with medical management, have a history of recurrent adenomas despite liver resection, have a rapid increase in the size and number of liver adenomas, and are at high risk for liver cancer.
After liver transplantation, all GSD I patients have achieved resolution of their metabolic derangement, including correction of hypoglycemia, lactic acidosis, hyperuricemia, and hyperlipidemia.
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