Results Summary and Professional Abstract
|This project's final research report is expected to be available by August 2020.|
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers asked why the researchers did not conduct biochemical assays of enzyme and metabolite levels or strive to correlate the levels of such markers to developmental outcomes. The researchers said they did not do such tests to avoid subjecting children in the study to a needle stick, which the researchers felt was not necessary. They used saliva to collect DNA to confirm the children’s diagnoses. The researchers noted that prior research implies that the children with Duarte galactosemia would all have normal levels of a key metabolite at their ages of 6 to 12, regardless of diet.
- The reviewers asked why the report did not discuss dietary exposure to galactose and not correlate galactose exposure to outcomes. The researchers said that they collected galactose exposure as a binary variable based on parent reports of their children’s diet when they were 2 to 12 months old. This enabled the largest number of cases to be available for comparison. However, the researchers noted that if they had seen any evidence of difference in outcomes between children who were exposed or not exposed to galactose, they would have attempted to analyze the issue further. Since no difference was observed, the researchers did not perform any further analysis.
Conflict of Interest Disclosures
View the COI disclosure form.