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Background Cardiovascular disease is the leading cause of death among patients with Duchenne muscular dystrophy (DMD). Identifying patients at risk of early death could allow for increased monitoring and more intensive therapy. Measures that associate with death could serve as surrogate outcomes in clinical trials. Methods and Results Duchenne muscular dystrophy subjects prospectively enrolled in observational studies were included. Models using generalized least squares were used to assess the difference of cardiac magnetic resonance measurements between deceased and alive subjects. A total of 63 participants underwent multiple cardiac magnetic resonance imaging and were included in the analyses. Twelve subjects (19.1%) died over a median follow‐up of 5 years (interquartile range, 3.1–7.0). Rate of decline in left ventricular ejection fraction was faster in deceased than alive subjects ( P <0.0001). Rate of increase in indexed left ventricular end‐diastolic ( P =0.0132) and systolic ( P <0.0001) volumes were higher in deceased subjects. Faster worsening in midcircumferential strain was seen in deceased subjects ( P =0.049) while no difference in global circumferential strain was seen. The rate of increase in late gadolinium enhancement, base T1, and mid T1 did not differ between groups. Conclusions Duchenne muscular dystrophy death is associated with the rate of change in left ventricular ejection fraction, midcircumferential strain, and ventricular volumes. Aggressive medical therapy to decrease the rate of progression may improve the mortality rate in this population. A decrease in the rate of progression may serve as a valid surrogate outcome for therapeutic trials.
Starnes et al. (Tue,) studied this question.