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满足患者友好型医学沟通的需求：对 2019 年 MPS VI 和 MPS IVA 管理建议的改编。

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

机构信息

Department of Paediatric Otolaryngology, Royal Manchester Children's Hospital, Manchester Academic Health Service (MAHSC), Manchester, UK.

Metabolism Unit, Department of Pediatrics, Gazi University, Ankara, Turkey.

出版信息

Orphanet J Rare Dis. 2022 Mar 2;17(1):91. doi: 10.1186/s13023-022-02219-7.

DOI:10.1186/s13023-022-02219-7
PMID:35236367
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8889702/
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.

摘要

背景

患者是任何疾病治疗中最重要的利益相关者，他们有教育需求，需要了解自己的病情和应接受的治疗。考虑到患者对聚焦于患者的材料的需求，我们提出了一种针对黏多糖贮积症（MPS）VI 和 MPS IVA 的定向方法，这两种疾病都是罕见的遗传性疾病，会影响多个器官和身体部位。最近发布了针对这些疾病治疗的独立指南，提供了基于证据和专业知识的建议，以优化患者管理。然而，尽管医疗保健提供者可能具备理解这些指南的培训和知识，但患者及其护理人员可能会发现这些技术内容具有挑战性。因此，我们旨在为 MPS VI 和 MPS IVA 指南开发以患者为主要受众的通俗易懂的摘要（PLS）。

结果

由专家团队对指南进行审查，确定了与患者相关的六个信息领域：多学科团队、定期检查和体检、疾病修正和支持性治疗、全身麻醉、耳鼻喉/呼吸护理以及手术。这些信息被改编成针对 MPS VI 或 MPS IVA 的一系列信息图，旨在吸引患者并以简洁明了的方式呈现信息。

结论

使用患者友好型材料，如我们开发的信息图，有可能更好地告知患者并使他们参与自己的治疗。我们向医学界发出“呼吁”，要求为旨在告知和增强患者能力的罕见病开发类似的 PLS 材料。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/4021c0c784ec/13023_2022_2219_Fig12_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/727c7c13c004/13023_2022_2219_Fig1_HTML.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/4021c0c784ec/13023_2022_2219_Fig12_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/727c7c13c004/13023_2022_2219_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/0ef74203ac2c/13023_2022_2219_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/4a63eb415b95/13023_2022_2219_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/f45c83bf3712/13023_2022_2219_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/a07508054fe2/13023_2022_2219_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/92da1e000a37/13023_2022_2219_Fig6_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/7d2a0f67f335/13023_2022_2219_Fig7_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/a83b93964465/13023_2022_2219_Fig8_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/a78574daf26d/13023_2022_2219_Fig9_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/867be1a01436/13023_2022_2219_Fig10_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/b302c17f25d0/13023_2022_2219_Fig11_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e12/8889702/4021c0c784ec/13023_2022_2219_Fig12_HTML.jpg

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Perceived burden in dealing with different rare diseases: a qualitative focus group study.应对不同罕见病时的感知负担：一项定性焦点小组研究
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A Community-Based Participatory Framework to Co-Develop Patient Education Materials (PEMs) for Rare Diseases: A Model Transferable across Diseases.基于社区的参与式框架，共同开发罕见病患者教育材料（PEMs）：一种可跨疾病转移的模式。
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