Priority 3 from the Liver Glycogen Storage Disease (International) PSP

UNCERTAINTY: How should optimal metabolic control both clinically and biochemically (like lactate, ketones and/or lipids) be achieved in liver Glycogen Storage Disease? (JLA PSP Priority 3)
Overall ranking 3
JLA question ID 0090/3
Explanatory note

The goal of treatment in liver GSD is to prevent metabolic derangement. There are several manners in which metabolic derangement can be prevented, such as dietary treatment.  


For details of the evidence checked, please see the spreadsheet held on the JLA website.

Health Research Classification System category  Metabolic and endocrine
Extra information provided by this PSP
Original uncertainty examples How important is it to keep ketone levels to a minimum? ~ prognostic markers for type IX? ~ What biochemical parameters are useful in the patient with GSD? How do you monitor the treatment in the patient to observe its effectiveness, as well as how to make appropiate adjustments in the treatment? ~ Why are there remaining strongly deviant metabolic parameters (lactate/triglycerides) even if there are no hypoglycemia's?
Submitted by Patient, carer and healthcare professionals
PSP information
PSP unique ID 0090
PSP name International Glycogen Storage Disease PSP
Total number of uncertainties identified by this PSP 72  (To see a full list of all uncertainties identified, please see the detailed spreadsheet held on the JLA website)
Date of priority setting workshop 24 May 2019