Results Summary and Professional Abstract
|This project's final research report is expected to be available by May 2021.|
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 expressed concern about engagement activities involving patient stakeholders the researchers recruited from among the study participants. The reviewers noted that these individuals tended to have short tenure as stakeholder participants because of their disease progression, which could indicate insufficient involvement in study decision-making by patient stakeholders. The researchers clarified that they had a large group of patient stakeholders who contributed to all aspects of study design and follow-up before the study began. The researchers explained that they recruited patient partners from among study participants to enhance the pool of stakeholders, but they were not the only patients or patient advocates among the stakeholders.
- The reviewers asked the researchers to address whether trying to create better risk stratification models is the best way to improve clinical decision-making for patients with acute myeloid leukemia (AML). The researchers added an analysis of the predictive power of their model to the report, saying that the model was clearly better than relying on physician perception after an initial clinic visit in predicting patient survival. The researchers said their work provided very specific survival rates for different risk groups, which did not exist before this study and which physicians can use to counsel patients. The researchers also stated that risk models can help improve decision making about treatments. The researchers also noted that their model was the first AML prognosis model to incorporate comorbidities.
- The reviewers asked for more justification for choosing one-year survival as the primary outcome and questioned whether it was the best outcome to consider especially for younger patients. The researchers agreed that younger patients may be interested in longer survival timepoints but said that their study largely focused on older patients and patients with comorbidities. The researchers also noted that when comparing more and less intensive therapies, they used two-year survival as an endpoint, and in one analysis they used all survival data accumulated over the more than four years since the study began.
Conflict of Interest Disclosures
The COI disclosure form for this project will be posted here soon.
Individuals with Rare Disease