International liver Glycogen Storage Disease PSP Question Verification Form

The purpose of the Question Verification Protocol is to enable Priority Setting Partnerships (PSPs) to clearly describe the process they employed to verify the uncertainty of their questions, before commending prioritisation. The template must be completed and submitted to the JLA with the questions template, for publication on the JLA website.

The JLA requires PSPs to be transparent and accountable in defining their own scope and process. This will enable researchers and other stakeholders to understand how individual PSPs have ascertained that their questions are unanswered, and any limitations therein.

Name of the PSP

International liver Glycogen Storage Disease Priority Setting Partnership (IGSDPSP)

Please describe the scope of the PSP

The scope of the International liver Glycogen Storage Disease PSP covers the care and/or management of liver Glycogen Storage Disease.
Inclusion criteria: The scope included all the work conducted by healthcare professionals and researchers that work in the field of liver Glycogen Storage Disease (GSD). Literature of all liver GSD types and all ages was included.
Exclusion criteria: We excluded literature not written in English.

Please provide a brief overview of your approach to verifying the uncertainty of your questions

The IGSDPSP has a broad scope including all aspects of care and/or management of liver GSD. We decided to include a very thorough search strategy to define literature in the evidence search since definitive evidence from meta-analyses or systematic reviews in the field of rare diseases is scarce.
When checking the evidence, first online sources have been reviewed. The choice was made to review the three main accessible databases for medical articles: Pubmed, Web of Science and Embase.
Search strategies based on Mesh terms and Title Abstract searches were made based on general terms and key words per summary question. Only articles relevant for liver Glycogen Storage Disease are mentioned in this document. If the question is not answered, the question remains the same. If the question is partially answered, a suggestion for adapting the question is made.
The evidence check was discussed in length with the steering group with regards to existing evidence and wording of the summary questions. In the end we reached full consensus on the evidence check.

Please list the type(s) of evidence you used to verify your questions as unanswered

Original articles (primary resources of original data), systematic reviews, national and international guidelines.

Please list the sources that you searched in order to identify that evidence

Pubmed, Web of Science and Embase.

What search terms did you use?

General terms: "Glycogen Storage Disease"[Mesh] OR "glycogen storage disease”[tiab]
Specific terms: defined as relevant Mesh- and title/abstract-terms of the key words per question.

Please describe the parameters of the search (eg time limits, excluded sources, country/language) and the rationale for any limitations

We included literature written in English. Articles before 2001 were only mentioned if evidence was scarce and if the articles were not referced in existing guidelines or systematic reviews.

Names of individuals who undertook the evidence checking

Willemijn Boonstra and Fabian Peeks (information specialists).

On what date was the question verification process completed?

December 13, 2018.

Any other relevant information

None.