Universidade Federal do Rio de Janeiro, Faculdade de Medicina, Rio de Janeiro RJ, Brazil.
Universidade Federal do Rio Grande do Sul, Faculdade de Medicina, Porto Alegre RS, Brazil.
Arq Neuropsiquiatr. 2023 Jan;81(1):81-94. doi: 10.1055/s-0043-1761466. Epub 2023 Mar 14.
In the last few decades, there have been considerable improvements in the diagnosis and care of Duchenne muscular dystrophy (DMD), the most common childhood muscular dystrophy. International guidelines have been published and recently reviewed. A group of Brazilian experts has developed a standard of care based on a literature review with evidence-based graded recommendations in a two-part publication. Implementing best practice management has helped change the natural history of this chronic progressive disorder, in which the life expectancy for children of the male sex in the past used to be very limited. Since the previous publication, diagnosis, steroid treatment, rehabilitation, and systemic care have gained more significant insights with new original work in certain fields. Furthermore, the development of new drugs is ongoing, and some interventions have been approved for use in certain countries. Therefore, we have identified the need to review the previous care recommendations for Brazilian patients with DMD. Our objective was to create an evidence-based document that is an update on our previous consensus on those topics.
在过去的几十年中,对杜氏肌营养不良症(DMD)的诊断和治疗有了相当大的改进,DMD 是最常见的儿童肌肉营养不良症。已经发布了国际指南,并最近进行了审查。一组巴西专家根据文献综述制定了护理标准,该标准以有证据支持的分级建议在两部分出版物中发布。实施最佳实践管理有助于改变这种慢性进行性疾病的自然病史,过去男性患儿的预期寿命非常有限。自上次发表以来,新的原始研究在某些领域为诊断、类固醇治疗、康复和全身护理提供了更多的重要见解。此外,新药物的开发正在进行中,一些干预措施已在某些国家获得批准使用。因此,我们认为有必要重新审查之前为巴西 DMD 患者制定的护理建议。我们的目标是创建一份基于证据的文件,对我们之前关于这些主题的共识进行更新。