Cardiorespiratory progression over 5 years and role of corticosteroids in DMD: a single site retrospective longitudinal study.

Cardiorespiratory progression over 5 years and role of corticosteroids in DMD: a single site retrospective longitudinal study. Chest. 2020 May 06;: Authors: Trucco F, Domingos J, Tay CG, Ridout D, Maresh K, Munot P, Sarkozy A, Robb S, Quinlivan R, Riley M, Burch M, Fenton M, Wallis C, Chan E, Abel F, Manzur A, Muntoni F Abstract BACKGROUND: Duchenne muscular dystrophy (DMD) boys treated with corticosteroids (CS) have prolonged survival and respiratory function when compared to CS-naïve. RESEARCH QUESTION: The differential impact of frequently used corticosteroids and their regimens on long-term (>5 years) cardiorespiratory progression in DMD children is unknown STUDY DESIGN: and Methods. Retrospective longitudinal study including DMD children followed at Dubowitz Neuromuscular Centre (Great Ormond Street Hospital London), May 2000-June 2017. Patients enrolled in any interventional clinical trials were excluded. We collected patients' anthropometrics, respiratory (forced vital capacity, FVC% predicted and absolute FVC, non-invasive ventilation requirement, NIV) and cardiac (left ventricular shortening function, LVFS%) function. CS-naïve patients had never received CS. CS-treated took either deflazacort or prednisolone, daily or intermittently (10 days on/10 days off) for >1 month. Average longitudinal models were fitted for yearly respiratory (FVC%P) and cardiac (LVFS%) progression. A time-to-event analysis to FVC%P<...
Source: Chest - Category: Respiratory Medicine Authors: Tags: Chest Source Type: research