Deflazacort use in non-ambulatory DMD patients resulted in slower average declines in FVC %-predicted vs. no steroids (+3.73 percentage points/year, p<0.05), with both steroids preserving function.
Cohort (n=86)
Does steroid treatment (prednisone or deflazacort) delay disease progression in non-ambulatory boys with Duchenne muscular dystrophy?
Steroid use (deflazacort or prednisone) in non-ambulatory patients with Duchenne muscular dystrophy is associated with delayed progression of pulmonary, cardiac, and upper extremity functional deficits compared to no steroids.
Effect estimate: +3.73 percentage points/year (FVC %-predicted, deflazacort vs no steroids)
p-value: p=<0.05
BACKGROUND: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is limited. OBJECTIVE: Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambulatory boys with DMD. METHODS: Disease progression was measured by functional status (Performance of Upper Limb Module for DMD 1.2 PUL and Egen Klassifikation Scale Version 2 EK scale) and by cardiac and pulmonary function (left ventricular ejection fraction LVEF, forced vital capacity FVC % -predicted, cough peak flow CPF). Longitudinal changes in outcomes, progression to key disease milestones, and dosing and body composition metrics were analyzed descriptively and in multivariate models. RESULTS: This longitudinal cohort study included 86 non-ambulatory patients with DMD (mean age 13.4 years; n = 40 deflazacort, n = 29 prednisone, n = 17 no steroids). Deflazacort use resulted in slower average declines in FVC % -predicted vs. no steroids (+3.73 percentage points/year, p < 0.05). Both steroids were associated with significantly slower average declines in LVEF, improvement in CPF, and slower declines in total PUL score and EK total score vs. no steroids; deflazacort was associated with slower declines in total PUL score vs. prednisone (all p < 0.05). Both steroids also preserved functional abilities considered especially important to quality of life, including the abilities to perform hand-to-mouth function and to turn in bed at night unaided (all p < 0.05 vs. no steroids). CONCLUSIONS: Steroid use after loss of ambulation in DMD was associated with delayed progression of important pulmonary, cardiac, and upper extremity functional deficits, suggesting some benefits of deflazacort over prednisone.
McDonald et al. (Mon,) conducted a cohort in Duchenne muscular dystrophy (n=86). Steroid treatment (prednisone or deflazacort) vs. No steroids was evaluated on Disease progression measured by functional status (PUL and EK scale) and by cardiac and pulmonary function (LVEF, FVC %-predicted, CPF) (+3.73 percentage points/year (FVC %-predicted, deflazacort vs no steroids), p=<0.05). Deflazacort use in non-ambulatory DMD patients resulted in slower average declines in FVC %-predicted vs. no steroids (+3.73 percentage points/year, p<0.05), with both steroids preserving function.