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Establishing a Core Outcome set for Mucopolysaccharidoses (MPS) in Children: Study Protocol for a Rapid Literature Review, Candidate Outcomes Survey, and Delphi Surveys

key information

source: Trials

year: 2021

authors: Howie AH,Tingley K,Inbar-Feigenberg M,Mitchell JJ,Butcher NJ,Offringa M,Smith M,Angel K,Gentle J,Wyatt A,Campeau PM,Chan A,Chakraborty P,El Turk F,Mamak E,Mhanni A,Skidmore B,Sparkes R,Stockler S,Potter BK,


Mucopolysaccharidoses (MPS) are a group of inherited metabolic diseases characterized by chronic, progressive multi-system manifestations with varying degrees of severity. Disease-modifying therapies exist to treat some types of MPS; however, they are not curative, underscoring the need to identify and evaluate co-interventions that optimize functioning, participation in preferred activities, and quality of life. A Canadian pediatric MPS registry is under development and may serve as a platform to launch randomized controlled trials to evaluate such interventions. To promote the standardized collection of patient/family-reported and clinical outcomes considered important to patients/families, health care providers (HCPs), and policymakers, the choice of outcomes to include in the registry will be informed by a core outcome set (COS). We aim to establish a patient-oriented COS for pediatric MPS using a multi-stakeholder approach.


organization: School of Epidemiology and Public Health, University of Ottawa, Room 101, 600 Peter Morand Crescent, Ottawa ON, Canada, Ottawa, ON, K1G 5Z3, Canada.

DOI: 10.1186/s13063-021-05791-8

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