Results Summary and Professional Abstract
|This project's final research report is expected to be available by March 2021.|
Related PCORI Dissemination and Implementation Project
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 commented on the lack of statistically significant results and warned the researchers not to attribute the lack of significance entirely to lower than expected sample size, since statistical significance in a larger sample was likely but might not be clinically meaningful. The reviewers cautioned against inferring too much from suggestive but not significant findings. The researchers acknowledged that it is tricky to convey an appropriate message from a study that failed to produce statistically significant results, but they felt that it would be a disservice to merely state that results were not significant and leave it at that. For example, they felt it was important to point out when large expected differences did not materialize, while for other parameters observed differences suggested clinically meaningful findings that can be tested in the future in larger, comparable samples. The researchers felt that it was important to lay out suggestive results and observations, with caveats about their significance, for a community in search of answers about best practices where there currently are none.
- The reviewers commented that the researchers did not achieve their third aim, which was to develop a strategy for disseminating findings. They suggested the researchers do more to come up with practical and useful applications of their findings. The researchers said the results of their quantitative analysis were complex, nuanced, and sometimes surprising. They expected to find more clinical benefits to liver transplantation than they did, but they said some of the challenges they faced, mainly that their two groups were not as comparable as they wished, reduced their ability to draw conclusions. The researchers explained that instead they would be collaborating with patients and families to determine the best ways to share study information with the community of patients with urea cycle disorders.
Conflict of Interest Disclosures
View the COI disclosure form.
^Mendel Tuchman, MD, was the original Principal Investigator on this project.