Final Research Report
View this project's final research report.
More to Explore...
DSC2U: Down Syndrome Clinic to You
Massachusetts General Hospital
Results of This Project
Stories and Videos
People with Down Syndrome Are Living Longer, but the Health System Still Treats Many as Kids
Tony Leys, KFF Health News, April 12, 2023
This article includes an interview with this study's Principal Investigator, Brian Skotko, MD, MPP, and highlights the clinical effectiveness of web-based platforms like Down Syndrome Clinic to You (DSC2U), which was created through this study to make it easier for people with Down Syndrome and their caregivers to access specialized information and expertise about the health services they need.
For People With Down Syndrome, the Best Medical Information is Now Automated
Genevieve Shaw Brown, ABC News, October 27, 2020
This article highlights DSC2U and features interviews with this study's Principal Investigator, Brian Skotko, MD, MPP, and Cristina Sanchez, one of the initial patients who were enrolled in DSC2U during the trial period.
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 question how the researchers treated missing data, given that there were 12 participants lost to follow-up in the intervention arm compared to 2 in the control arm. The researchers admitted that while they had intended to count these participants as failing to complete the evaluation, this was not done and not considered in the final analyses. When the researchers reran the analyses including the missing cases as incomplete evaluations, they found that the intervention still had significant benefit over the control in participant families completing indicated evaluations. However, this more conservative analysis strategy found that the intervention was not statistically superior to the control in participant families completing nonindicated evaluations.
- The reviewers cautioned the researchers not to overstate the significance of the relative difference in risk ratio between the intervention and control conditions, when the absolute difference in risk ratio in completed evaluations did not reach the clinically meaningful difference established at the beginning of the study. The researchers acknowledged this point but stated that there are instances where the relative difference in risk ratio is more relevant for clinical decisions. Therefore, the researchers presented both risk ratios in the report so that readers could evaluate the significance of the study on both scales.
- The reviewers recommended including the results from the more complicated statistical procedures the researchers used in testing the primary outcomes. The researchers added the significance values for the more complicated tests but chose to not include more of those results because they felt that the t-test results best conveyed the results of the trial since they used a scale that was more meaningful clinically.
Conflict of Interest Disclosures
Patient / Caregiver Partners
- Sandra Baker
- Patricia Bauer
- Jawanda Mast
- Missy Skalvem
- Ben Majewski,
- Maureen Gallagher and Sarah Cullen of the Massachusetts Down Syndrome Congress
Other Stakeholder Partners
- Dr. Ariel Frey
- Dr. Travis Riddell
- Dr. Ashley Lamb
- Dr. Meghan Kiefer
- Dr. Melissa Parisi
- Dr. Marilyn Bull
- Dr. Brian Chicoine
- Dr. Karen Sepucha
- Has Results