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

Iain A. Bruce, Fatih S. Ezgü, Christoph Kampmann, Vladimir Kenis, William Mackenzie, Bob Stevens, Robert Walker, Christian Hendriksz

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Article number91
JournalOrphanet Journal of Rare Diseases
Volume17
Issue number1
DOIs
Publication statusPublished - Dec 2022

Keywords

  • Anesthetics
  • Enzyme replacement therapy
  • Management guidelines
  • MPS IVA
  • MPS VI
  • Mucopolysaccharidosis
  • Patient education
  • Patient engagement
  • Rare diseases
  • Surgery

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