Benefícios de longo prazo e os efeitos adversos doscorticóides comparando o esquema intermitente contra o esquema diário na distrofia muscular de Duchenne
Reino Unido – os autores acompanharam por 4 anos 360 meninos com Duchenne em tratamento com prednisolona comparando o uso de doses diárias da medicação comparando com o uso do esquema intermintente (10 dias com e 10 dias sem). Houve mlehora em ambos os grupos mas o que receberam doses diárias conseguiram deambular por mais tempo mas tiveram maior frequência de efeitos colaterais. Ou seja o uso de corticóides na distrofia muscular de Duchenne é mandatório.
O resumo em inglês pode ser lido abaixo:
(J Neurol Psychiatry, Dec 2012) Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
Valeria Ricotti, Deborah A Ridout, Elaine Scott, Ros Quinlivan, Stephanie A Robb, Adnan Y Manzur, Francesco Muntoni, on behalf of the NorthStar Clinical Network – UK
Objective To assess the current use of glucocorticoids (GCs) in Duchenne muscular dystrophy in the UK, and compare the benefits and the adverse events of daily versus intermittent prednisolone regimens.
Design A prospective longitudinal observational study across 17 neuromuscular centres in the UK of 360 boys aged 3–15 years with confirmed Duchenne muscular dystrophy who were treated with daily or intermittent (10days on/10days off) prednisolone for a mean duration of treatment of 4years
Results The median loss of ambulation was 12years in intermittent and 14.5years in daily treatment; the HR for intermittent treatment was 1.57 (95% CI 0.87 to 2.82). A fitted multilevel model comparing the intermittent and daily regiments for the NorthStar Ambulatory Assessment demonstrated a divergence after 7 years of age, with boys on an intermittent regimen declining faster (p<0.001). Moderate to severe side effects were more commonly reported and observed in the daily regimen, including Cushingoid features, adverse behavioural events and hypertension. Body mass index mean z score was higher in the daily regimen (1.99, 95% CI 1.79 to 2.19) than in the intermittent regimen (1.51, 95% CI 1.27 to 1.75). Height restriction was more severe in the daily regimen (mean z score −1.77, 95% CI −1.79 to −2.19) than in the intermittent regimen (mean z score −0.70, 95% CI −0.90 to −0.49).
Conclusions Our study provides a framework for providing information to patients with Duchenne muscular dystrophy and their families when introducing GC therapy. The study also highlights the importance of collecting longitudinal natural history data on patients treated according to standardised protocols, and clearly identifies the benefits and the side-effect profile of two treatment regimens, which will help with informed choices and implementation of targeted surveillance.
