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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.
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RESUMEN Objetivo : Elaborar una guía de práctica clínica peruana para el diagnóstico y tratamiento de la Distrofia Muscular de Duchenne y Becker (DMD). Materiales y métodos : Se conformó un grupo elaborador de la guía (GEG) que incluyó médicos especialistas en neurología, neuropediatría, genética y metodología. El GEG formuló ocho preguntas para desarrollar las recomendaciones de la Guía de Práctica Clínica (GPC). Se realizó una búsqueda sistemática en Medline, Scopus y CCRT durante el periodo enero-abril 2021 para responder a las preguntas PICO. La certeza de la evidencia fue evaluada usando la metodología Grading of Recommendations Assessment, Development, and Evaluation (GRADE). Resultados : Las preguntas PICO, se orientaron para explorar el tamizaje, diagnóstico y tratamiento de la DMD. Se formularon 15 recomendaciones (10 fuertes, 5 condicionales) y 11 puntos de buena práctica clínica Conclusión : Se presenta la guía para el diagnóstico y tratamiento de la DMD, elaborada bajo una metodología basada en las evidencias actuales.
ABSTRACT Objective : to provide evidence-based clinical recommendations for the diagnosis and treatment of Duchenne Muscular Dystrophy. Methods : a guideline development group (GEG) was formed that included specialized physicians in the fields of neurology, neuropediatrics, genetics, and methodology. The GEG asked eight clinical questions to be answered by recommendations in this clinical practice guidelines (CPG). We conducted a systematic search and - when deemed relevant - primary studies in Medline, Scopus, and the Cochrane Controlled Register of Trials during 2021 were reviewed. Evidence was selected to answer each of the clinical questions posed. Certainty of the evidence was assessed using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) system. In periodic work meetings, the GEG used the GRADE methodology to review the evidence and formulate recommendations, points of good clinical practice, and a diagnosis and treatment flowchart. Results : this CPG addressed eight clinical questions, divided into three topics: screening, diagnosis, and treatment. Based on these questions, fifteen recommendations were formulated (10 strong, 5 conditional) and 11 points for good clinical practice. Conclusion : this paper summarizes the methodology and evidence- based conclusions of the CPG for the diagnosis and treatment of Duchenne muscular dystrophy.