作者
Michael Pugliese, Kylie Tingley, Andrea Chow, Nicole Pallone, Maureen Smith, Pranesh Chakraborty, Michael T Geraghty, Julie K Irwin, John J Mitchell, Sylvia Stockler, Stuart G Nicholls, Martin Offringa, Alvi Rahman, Laure A Tessier, Nancy J Butcher, Ryan Iverson, Monica Lamoureux, Tammy J Clifford, Brian Hutton, Karen Paik, Jessica Tao, Becky Skidmore, Doug Coyle, Kathleen Duddy, Sarah Dyack, Cheryl R Greenberg, Shailly Jain Ghai, Natalya Karp, Lawrence Korngut, Jonathan Kronick, Alex MacKenzie, Jennifer MacKenzie, Bruno Maranda, Murray Potter, Chitra Prasad, Andreas Schulze, Rebecca Sparkes, Monica Taljaard, Yannis Trakadis, Jagdeep Walia, Beth K Potter, Canadian Inherited Metabolic Diseases Research Network
发表日期
2021/8/1
期刊
Pediatrics
卷号
148
期号
2
出版商
American Academy of Pediatrics
简介
BACKGROUND
Evidence to guide treatment of pediatric medium-chain acyl-coenzyme A dehydrogenase (MCAD) deficiency and phenylketonuria (PKU) is fragmented because of large variability in outcome selection and measurement. Our goal was to develop core outcome sets (COSs) for these diseases to facilitate meaningful future evidence generation and enhance the capacity to compare and synthesize findings across studies.
METHODS
Parents and/or caregivers, health professionals, and health policy advisors completed a Delphi survey and participated in a consensus workshop to select core outcomes from candidate lists of outcomes for MCAD deficiency and PKU. Delphi participants rated the importance of outcomes on a nine-point scale (1–3: not important, 4–6: important but not critical, 7–9: critical). Candidate outcomes were progressively narrowed down over 3 survey rounds. At the workshop …
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M Pugliese, K Tingley, A Chow, N Pallone, M Smith… - Pediatrics, 2021