• Libraries
    • Log in to:
    View Item 
    •   MSpace Home
    • University of Manitoba Researchers
    • University of Manitoba Scholarship
    • View Item
    •   MSpace Home
    • University of Manitoba Researchers
    • University of Manitoba Scholarship
    • View Item
    JavaScript is disabled for your browser. Some features of this site may not work without it.

    Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys

    Thumbnail
    View/Open
    13063_2021_Article_5791.pdf (1.162Mb)
    Date
    2021-11-17
    Author
    Howie, Alison H.
    Tingley, Kylie
    Inbar-Feigenberg, Michal
    Mitchell, John J.
    Butcher, Nancy J.
    Offringa, Martin
    Smith, Maureen
    Angel, Kim
    Gentle, Jenifer
    Wyatt, Alexandra
    Campeau, Philippe M.
    Chan, Alicia
    Chakraborty, Pranesh
    El Turk, Farah
    Mamak, Eva
    Mhanni, Aizeddin
    Skidmore, Becky
    Sparkes, Rebecca
    Stockler, Sylvia
    Potter, Beth K.
    Metadata
    Show full item record
    Abstract
    Abstract Background 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. Methods In step 1 of the six-step process to develop the COS, we will identify relevant outcomes through a rapid literature review and candidate outcomes survey. A two-phase screening approach will be implemented to identify eligible publications, followed by extraction of outcomes and other pre-specified data elements. Simultaneously, we will conduct a candidate outcomes survey with children with MPS and their families to identify outcomes most important to them. In step 2, HCPs experienced in treating patients with MPS will be invited to review the list of outcomes generated in step 1 and identify additional clinically relevant outcomes. We will then ask patients/families, HCPs, and policymakers to rate the outcomes in a set of Delphi Surveys (step 3), and to participate in a subsequent consensus meeting to finalize the COS (step 4). Step 5 involves establishing a set of outcome measurement instruments for the COS. Finally, we will disseminate the COS to knowledge users (step 6). Discussion The proposed COS will inform the choice of outcomes to include in the MPS registry and, more broadly, promote the standardized collection of patient-oriented outcomes for pediatric MPS research. By involving patients/families from the earliest stage of the research, we will ensure that the COS will be relevant to those who will ultimately benefit from the research. Trial registration PROSPERO CRD42021267531 , COMET
    URI
    https://doi.org/10.1186/s13063-021-05791-8
    http://hdl.handle.net/1993/36131
    Collections
    • Rady Faculty of Health Sciences Scholarly Works [1296]
    • University of Manitoba Scholarship [1978]

    DSpace software copyright © 2002-2016  DuraSpace
    Contact Us | Send Feedback
    Theme by 
    Atmire NV
     

     

    Browse

    All of MSpaceCommunities & CollectionsBy Issue DateAuthorsTitlesSubjectsThis CollectionBy Issue DateAuthorsTitlesSubjects

    My Account

    Login

    Statistics

    View Usage Statistics

    DSpace software copyright © 2002-2016  DuraSpace
    Contact Us | Send Feedback
    Theme by 
    Atmire NV