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Core outcome set for pediatric chronic pain clinical trials: results from a Delphi poll and consensus meeting
Tonya M Palermo1,2, Gary A Walco2, Unmesha Roy Paladhi3
1Center for Child Health, Behavior, and Development, Seattle Children's Research Institute, Seattle, WA, United States.
Insights
This study established a core outcome set for pediatric chronic pain interventions. Mandatory domains include pain severity, pain interference, well-being, and adverse events for better clinical trials.
Area of Science:
- Pediatric Pain Management
- Clinical Trial Methodology
- Outcome Measurement
Background:
- Advancing care for children with chronic pain requires high-quality intervention trials and appropriate outcome measures.
- A standardized core outcome set is needed for pediatric chronic pain interventions.
Purpose of the Study:
- To update a core outcome set for pediatric chronic pain interventions.
- To identify and reach consensus on mandatory and optional outcome domains for clinical trials.
Main Methods:
- A modified Delphi study involving 3 stakeholder groups: children with chronic pain (n=93), parents (n=90), and healthcare providers (n=52).
- Two rounds of surveys followed by a virtual consensus conference.
- Quantitative and qualitative data analysis to identify and rank outcome domains.
Main Results:
- Ten outcome domains were identified from stakeholder input.
- Pain severity, pain interference with daily living, overall well-being, and adverse events (including death) were unanimously voted as mandatory domains.
- Emotional functioning, physical functioning, and sleep were identified as important optional domains.
Conclusions:
- A consensus-based core outcome set was established for pediatric chronic pain clinical trials.
- The defined mandatory domains will ensure consistent assessment across interventions.
- Future research should focus on selecting validated measures for these outcome domains and exploring emerging areas like biomarkers.
Abstract:
Appropriate outcome measures and high-quality intervention trials are critical to advancing care for children with chronic pain. Our aim was to update a core outcome set for pediatric chronic pain interventions. The first phase involved collecting providers', patients', and parents' perspectives about treatment of pediatric chronic pain to understand clinically meaningful outcomes to be routinely measured. The second phase was to reach consensus of mandatory and optional outcome domains following the OMERACT framework. A modified Delphi study with 2 rounds was conducted including 3 stakeholder groups: children with chronic pain (n = 93), their parents (n = 90), and health care providers who treat youth with chronic pain (n = 52). Quantitative and qualitative data from round 1 of the Delphi study were summarized to identify important outcomes, which were condensed to a list of 10 outcome domains. Round 2 surveys were analyzed to determine the importance of the 10 domains and their relative ranking in each stakeholder group. A virtual consensus conference was held with the steering committee to reach consensus on a set of recommended outcome domains for pediatric chronic pain clinical trials. It was determined, by unanimous vote, that pain severity, pain interference with daily living, overall well-being, and adverse events, including death, would be considered mandatory domains to be assessed in all trials of any type of intervention. Emotional functioning, physical functioning, and sleep were important but optional domains. Last, the research agenda identifies several important emerging areas, including biomarkers. Future work includes selecting appropriate validated measures to assess each outcome domain.
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