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杜氏肌营养不良症诊断与管理的临床实践指南:一项范围综述

Clinical practice guidelines for the diagnosis and management of Duchenne muscular dystrophy: a scoping review.

作者信息

Malaga Marco, Rodriguez-Calienes Aaron, Chavez-Ecos Fabian A, Huerta-Rosario Andrely, Alvarado-Gamarra Giancarlo, Cabanillas-Lazo Miguel, Moran-Ballon Paula, Velásquez-Rimachi Victor, Martinez-Esteban Peggy, Alva-Diaz Carlos

机构信息

Facultad de Medicina Humana de la Universidad de San Martín de Porres, Lima, Peru.

Red de Eficacia Clínica y Sanitaria, REDECS, Lima, Peru.

出版信息

Front Neurol. 2024 Jan 5;14:1260610. doi: 10.3389/fneur.2023.1260610. eCollection 2023.

Abstract

INTRODUCTION

Our objective was to identify recent CPGs for the diagnosis and management of DMD and summarize their characteristics and reliability.

METHODS

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.

RESULTS

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.

DISCUSSION

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

引言

我们的目标是识别最近关于杜氏肌营养不良症(DMD)诊断和管理的临床实践指南(CPGs),并总结其特点和可靠性。

方法

我们使用MEDLINE、转化研究为实践(TRIP)数据库、谷歌学术、各组织制定的指南以及其他知识库对CPGs进行了范围综述,以识别过去5年发表的CPGs。我们的方案是根据系统评价和Meta分析的首选报告项目制定的范围综述。为了评估CPGs的可靠性,我们使用了指南研究与评价II(AGREE II)中包含的所有领域。

结果

我们选择了2015年至2020年间发表或更新的三份CPGs。所有指南都显示出良好或足够的方法严谨性,但在利益相关者参与和适用性领域存在缺陷。除了给药方案上的细微差异外,CPGs在类固醇治疗方面的建议是一致的。然而,对于新药的建议则有所不同。

讨论

需要有最新且可靠的CPGs,这些指南要涵盖DMD管理的广泛主题,并考虑为罕见病制定建议时面临的挑战。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f32f/10797703/0545f92e83f9/fneur-14-1260610-g0001.jpg

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