No abstract

Significant advances in the understanding and management of Duchenne muscular dystrophy (DMD) have occurred since the publication of international guidelines for DMD care in 2010. Our objective was to provide an evidence-based national consensus statement for multidisciplinary care of DMD in Brazil. A combination of the Delphi technique with a systematic review of studies from 2010 to 2016 was employed to classify evidence levels and grade of recommendations for the guideline. Our recommendations were divided in two parts. Guideline methodology and overall disease concept descriptions are found in Part 1. Here we present Part 2, where we provide the results and recommendations on rehabilitation and systemic care for DMD.

A paralisia cerebral é uma sequela de agressão encefálica caracterizada por transtorno motor não evolutivo quanto à sua lesão. De acordo com o local da lesão pode provocar diferentes seqüelas que resultam em incapacidade funcional. OBJETIVO: revisar de forma não sistemática sobre o tema paralisia cerebral, enfocando o seu prognóstico motor, incluindo expectativa de vida e funcionalidade. MÉTODO: utilização das bases de dados Medline e LILACS, dos últimos quinze anos, com os unitermos paralisia cerebral, quadriplegia, diplegia, hemiplegia, prognóstico. RESULTADOS: foram listados 474 artigos científicos, sendo selecionados para esta revisão 34 artigos com base em: título, resumo, assunto, originalidade e disponibilidade de acesso pelo portal da CAPES. Foram identificadas as variáveis que podem influenciar o prognóstico motor nas crianças com paralisia cerebral. O prejuízo motor das crianças com paralisia cerebral está diretamente relacionado com o nível de gravidade da paralisia cerebral, assistência terapêutica e o tipo apresentado pelas crianças. Os pacientes com hemiplegia resultante de paralisia cerebral apresentam um prognóstico motor mais favorável comparado aos diplégicos e quadriplégicos.

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.