Key result
LV dysfunction onset before age 18 is linked to death ~12 years earlier than adult onset.
Cohort (n=57)
No
Absolute Event Rate: 21.1% vs 33.1%
p-value: p=<0.001
In patients with Duchenne muscular dystrophy receiving contemporary therapies, left ventricular dysfunction progresses almost universally, and onset before age 18 is associated with significantly shortened survival.
Earlier LV dysfunction onset associated with shorter survival in DMD; extends observations but leaves optimal management open.
Objective: To describe the natural history of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD) who are receiving contemporary therapies. Methods: This is a single-institution retrospective cohort study of 57 patients aged >15 years with DMD. Serial digital echocardiograms were performed over a median follow-up of 8 years. Left ventricular dysfunction (LVD) was defined as shortening fraction (SF) <29% plus focal wall motion abnormalities. Therapies included ACE inhibitors, beta-blockers and assisted ventilation. Results: The SF declined progressively in 53/57 patients (93%). LVD occurred in 40 of 57 patients (70%), with variable age at onset (median 18 years, IQR 14-21.5 years). Rate of SF decline (-1.51%±1.16%/year) was variable and unrelated to genotype. However, survival was shorter for patients with LVD onset at age <18 years vs onset at ≥18 years (death at 21.1±2.5 years vs 33.1±4.4 years; P<0.001). Death occurred in 27/57 (47%) patients at a median age of 26.3 years (IQR 20.6-31.5). Death was preceded by LVD in 22/27 patients (81%), 15 (68%) of whom developed class 4 heart failure (CHF). Time from CHF to death was brief (median 8.0 months). Conclusion: Despite contemporary therapies, SF declined progressively in almost all patients. Age at onset of LVD and age at death were variable and unrelated to genotype; however, survival was shortened for patients with LVD onset at age <18 years. Death was usually preceded by LVD. CHF was a sentinel event, with death occurring shortly thereafter.
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Wang et al. (2018) conducted a cohort in Duchenne muscular dystrophy (DMD) (n=57). Left ventricular dysfunction (LVD) onset at age <18 years vs. LVD onset at ≥18 years was evaluated on Age at death (p=<0.001). Left ventricular dysfunction onset at age <18 years was associated with significantly earlier death compared to onset at ≥18 years (21.1 vs 33.1 years; P<0.001).
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