Core Outcome Sets for Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Phenylketonuria.
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Pugliese Michael, Tingley Kylie, Chow Andrea, Pallone Nicole, Smith Maureen, Chakraborty Pranesh, Geraghty Michael T, Irwin Julie K, Mitchell John J, Stockler Sylvia, Nicholls Stuart G, Offringa Martin, Rahman Alvi, Tessier Laure A, Butcher Nancy J, Iverson Ryan, Lamoureux Monica, Clifford Tammy J, Hutton Brian, Paik Karen, Tao Jessica, Skidmore Becky, Coyle Doug, Duddy Kathleen, Dyack Sarah, Greenberg Cheryl R, Jain Ghai Shailly, Karp Natalya, Korngut Lawrence, Kronick Jonathan, MacKenzie Alex, MacKenzie Jennifer, Maranda Bruno, Potter Murray, Prasad Chitra, Schulze Andreas, Sparkes Rebecca, Taljaard Monica, Trakadis Yannis, Walia Jagdeep, Potter Beth K, Canadian Inherited Metabolic Diseases Research Network
Аннотация
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, participants evaluated the remaining candidate outcomes using an adapted nominal technique, open discussion, and voting. After the workshop, we finalized the COSs and recommended measurement instruments for each outcome.
RESULTS: There were 85, 61, and 53 participants across 3 Delphi rounds, respectively. The candidate core outcome lists were narrowed down to 20 outcomes per disease to be discussed at the consensus workshop. Voting by 18 workshop participants led to COSs composed of 8 and 9 outcomes for MCAD deficiency and PKU, respectively, with measurement recommendations.
CONCLUSIONS: These are the first known pediatric COSs for MCAD deficiency and PKU. Adoption in future studies will help to ensure best use of limited research resources to ultimately improve care for children with these rare diseases.