关键词: Duchenne muscular dystrophy evidence-based practice practice guidelines rare diseases review

来  源:   DOI:10.3389/fneur.2023.1260610   PDF(Pubmed)

Abstract:
UNASSIGNED: Our objective was to identify recent CPGs for the diagnosis and management of DMD and summarize their characteristics and reliability.
UNASSIGNED: We conducted a scoping review of CPGs using MEDLINE, the Turning Research Into Practice (TRIP) database, Google Scholar, guidelines created by organizations, and other repositories to identify CPGs published in the last 5 years. Our protocol was drafted using the Preferred Reporting Items for Systematic Reviews and Meta-analyses for scoping reviews. To assess the reliability of the CPGs, we used all the domains included in the Appraisal of Guidelines Research and Evaluation II.
UNASSIGNED: We selected three CPGs published or updated between 2015 and 2020. All the guidelines showed good or adequate methodological rigor but presented pitfalls in stakeholder involvement and applicability domains. Recommendations were coherent across CPGs on steroid treatment, except for minor differences in dosing regimens. However, the recommendations were different for new drugs.
UNASSIGNED: There is a need for current and reliable CPGs that develop broad topics on the management of DMD and consider the challenges of developing recommendations for RDs.
摘要:
我们的目标是确定用于DMD诊断和管理的最新CPG,并总结其特征和可靠性。
我们使用MEDLINE对CPG进行了范围审查,将研究转化为实践(TRIP)数据库,谷歌学者,组织创建的指导方针,和其他存储库,以识别过去5年发布的CPG。我们的方案是使用系统审查的首选报告项目和范围审查的荟萃分析起草的。为了评估CPG的可靠性,我们使用了评估指南研究与评估II中包含的所有领域。
我们选择了三个在2015年至2020年之间发布或更新的CPG。所有指南都显示出良好或足够的方法严谨性,但在利益相关者的参与和适用性领域存在陷阱。关于类固醇治疗的建议在CPG中是一致的,除了给药方案的微小差异。然而,对于新药,建议是不同的。
需要当前可靠的CPG,以开发有关DMD管理的广泛主题,并考虑为RD开发建议的挑战。
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