Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA

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dc.contributor.author Bruce, Iain A.
dc.contributor.author Ezgu, Fatih S.
dc.contributor.author Kampmann, Christoph
dc.contributor.author Kenis, Vladimir
dc.contributor.author Mackenzie, William
dc.contributor.author Stevens, Bob
dc.contributor.author Walker, Robert
dc.contributor.author Hendriksz, Christian J.
dc.date.accessioned 2023-07-14T11:19:24Z
dc.date.available 2023-07-14T11:19:24Z
dc.date.issued 2022-03
dc.description.abstract BACKGROUND : Patients are the most important stakeholders in the care of any disease and have an educational need to learn about their condition and the treatment they should receive. Considering this need for patient-focused materials, we present a directed approach for mucopolysaccharidosis (MPS) VI and MPS IVA, a pair of rare, inherited diseases that affects multiple organs and parts of the body. Independent guidelines on the treatment of these diseases were recently published, providing evidence- and expertise-driven recommendations to optimize patient management. However, while healthcare providers may have the training and knowledge to understand these guidelines, patients and their caregivers can find the technical content challenging. Hence, we aimed to develop plain language summaries (PLS) of the MPS VI and MPS IVA guidelines with patients as the primary audience. RESULTS : A review of the guidelines by an expert team identified six domains of information relevant to patients: The multidisciplinary team, regular tests and check-ups, disease-modifying and supportive treatments, general anesthetics, ear-nose-throat/respiratory care, and surgeries. This information was adapted into a series of infographics specific to either MPS VI or MPS IVA, designed to appeal to patients and clearly present information in a concise manner. CONCLUSIONS : The use of patient-friendly materials, like the infographics we have developed, has the potential to better inform patients and engage them in their care. We issue a “call to arms” to the medical community for the development of similar PLS materials in rare diseases intended to inform and empower patients. en_US
dc.description.department Paediatrics and Child Health en_US
dc.description.librarian hj2023 en_US
dc.description.sponsorship BioMarin. en_US
dc.description.uri https://ojrd.biomedcentral.com en_US
dc.identifier.citation Bruce, I.A., Ezgü, F.S., Kampmann, C. et al. Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA. Orphanet Journal of Rare Diseases 17, 91 (2022). https://doi.org/10.1186/s13023-022-02219-7. en_US
dc.identifier.issn 1750-1172
dc.identifier.other 10.1186/s13023-022-02219-7
dc.identifier.uri http://hdl.handle.net/2263/91459
dc.language.iso en en_US
dc.publisher BioMed Central en_US
dc.rights © The Author(s) 2022. Open Access. This article is licensed under a Creative Commons Attribution 4.0 International License. en_US
dc.subject Mucopolysaccharidosis (MPS) en_US
dc.subject Rare diseases en_US
dc.subject MPS IVA en_US
dc.subject MPS VI en_US
dc.subject Management guidelines en_US
dc.subject Enzyme replacement therapy en_US
dc.subject Surgery en_US
dc.subject Anesthetics en_US
dc.subject Patient engagement en_US
dc.subject Patient education en_US
dc.subject SDG-03: Good health and well-being en_US
dc.title Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA en_US
dc.type Article en_US


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