Symptomatic Presentation Influences Outcomes in Pediatric Restrictive Cardiomyopathy

Children with restrictive cardiomyopathy (RCM) traditionally have a poor prognosis, with most patients either dying or requiring heart transplantation within 2 years of diagnosis. The development of symptoms in RCM suggests advanced disease, however no study has directly compared outcomes of these patients based on symptoms at time of diagnosis. The purpose of this retrospective cohort study was to compare children with RCM presenting with symptoms to those presenting without symptoms. Times to transplantation, death, and a composite outcome of adverse cardiac events (CPR, cardioversion, inotropic support, mechanical ventilation, mechanical support, or heart transplant) were outcomes of interest for statistical analysis. In total 25 patients with RCM were included. At 2 years following diagnosis, asymptomatic patients had a significantly better transplant-free survival at 57% compared to 17% for symptomatic patients (p = 0.03). Asymptomatic patients also had significantly improved cardiac event-free survival at 71% compared to symptomatic patients at 25% (p = 0.01). In multivariable analysis, cardiac symptoms at presentation remained an independent risk factor for heart-transplant or death (hazard ratio 5.17 (1.28–20.85), p = 0.02). In conclusion, the development of cardiac symptoms in pediatric RCM patients may be associated with a worse transplant-free survival.