Evolução da função cardíaca e mortalidade em pacientes com Duchenne com e seu o uso de corticóides
Canadá – 86 pacientes com Duchenne foram acompanhados, sendo 63 usando corticóides e 23 sem o tratamento. A mortalidade foi 76% maior no período estudado entre os que não usavam corticóides. A perda de função cardíaca e as manifestações de cardiomiopatia foram menores entre os tratados. Os autores concluiram que o uso de corticoides na distrofia muscular de Duchenne está associado a uma redução substancial da mortalidade por qualquer causa e cardiomiopatia de início recente e progressiva.
O resumo em inglês e o editorial do artigo podem ser lidos abaixo:
(Journal of the American College of Cardiology, 2013) All-Cause Mortality and Cardiovascular Outcomes With Prophylactic Steroid Therapy in Duchenne Muscular Dystrophy
Gernot Schram, Anne Fournier, Hugues Leduc, ,Nagib Dahdah, Johanne Therien, Michel Vanasse, Paul Khairy – Canada
Objectives The aim of this study was to determine the impact of steroid therapy on cardiomyopathy and mortality in patients with Duchenne muscular dystrophy (DMD).
Background DMD is a debilitating X-linked disease that afflicts as many as 1 in 3,500 boys. Although steroids slow musculo skeletal impairment, the effects on cardiac function and mortality remain unknown.
Methods We conducted a cohort study on patients with DMD treated with renin-angiotensin-aldosterone system antagonists with or without steroid therapy.
Results Eighty-six patients, 9.1 + 3.5 years of age, were followed for 11.3 + 4.1 years. Seven of 63 patients (11%) receiving steroid therapy died compared with 10 of 23 (43%) not receiving steroid therapy (p <0.0010). Overall survival rates at 5, 10, and 15 years of follow-up were 100%, 98.0%, and 78.6%, respectively, for patients receiving steroid therapy versus 100%, 72.1%, and 27.9%, respectively, for patients not receiving steroid therapy (log-rank p< 0.0005). In multivariate propensity-adjusted analyses, steroid use was associated with a 76% lower mortality rate (hazard ratio: 0.24; 95% confidence interval: 0.07 to 0.91; p 0.0351). The mortality reduction was driven by fewer heart failure–related deaths (0% vs. 22%, p 0.0010). In multivariate analyses, steroids were associated with a 62% lower rate of new-onset cardiomyopathy (hazard ratio: 0.38; 95% confidence interval: 0.16 to 0.90; p 0.0270). Annual rates of decline in left ventricular ejection fraction ( 0.43% vs. 1.09%, p 0.0101) and shortening fraction ( 0.32% vs. 0.65%, p 0.0025) were less steep in steroid treated patients. Consistently, the increase in left ventricular end-diastolic dimension was of lesser magnitude ( 0.47 vs. 0.92 mm per year, p 0.0105).
Conclusions In patients with DMD, steroid therapy is associated with a substantial reduction in all-cause mortality and new-onset and progressive cardiomyopathy.
Editorial: Steroid Therapy Effectively Delays Duchenne’s Cardiomyopathy
