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

ABSTRACT Background Duchenne muscular dystrophy (DMD) boys treated with corticosteroids (CS) have prolonged survival and respiratory function when compared to CS-naive. 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-naive 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 Results There were 270 patients, mean age at baseline 6.2 (±2.3) years. Median follow-up 5.6 (± 3.5) years. At baseline, 263 were ambulant. Sixty-six were CS-daily, 182 CS-intermittent >60% treatment, 22 CS-naive. Yearly FVC%P declined similarly from 9 years (5.9% and 6.9%/year, p=0.27) in CS-daily and CS-intermittent. CS-daily declined from a higher FVC%P than CS-intermittent (p 2 years later than CS-treated. LVFS% declined by 0.53%/year in CS-treated irrespective of CS regimen, significantly slower (p Interpretation. CS irrespective of their regimen significantly improved respiratory function and delayed NIV requirement and cardiomyopathy.