Management of children with dilated cardiomyopathy in The Netherlands: Implications of a low early transplantation rate.

BACKGROUND: The policy for listing and transplant for children with dilated cardiomyopathy (DCM) in The Netherlands has been conservative because of low donor availability. The effects of this policy on outcome are reported. METHODS: This was a multicenter, nationwide study performed in 148 children with DCM. The primary outcome was death or heart transplant. RESULTS: Overall, 43 patients (29%) died or were transplanted. Within 1 year of diagnosis, 21 patients died, and only 4 underwent transplantation (3 on mechanical circulatory support). The 1-year survival was 85% (95% confidence interval [CI] = 79-91), and 5-year survival was 84% (95% CI = 78-90). Transplantation-free survival at 1 year was 82% (95% CI = 75-88) and at 5 years was 72% (95% CI = 64-80). Within 1 year of diagnosis, with death as the main end-point (21 of 25, 84%), intensive care unit admission (hazard ratio = 2.6, p = 0.05) and mechanical circulatory support (hazard ratio = 3.2, p = 0.03) were risk factors (multivariable Cox analysis); inotropic support was longer in patients reaching an end-point. At >1 year after diagnosis, with transplantation as the main end-point (15 of 18, 83%), age >6 years (hazard ratio = 6.1, p = 0.02) was a risk factor. There were 56 (38%) children who recovered, 50% within 1 year of diagnosis. Recovery was associated with younger age; was similar in patients with myocarditis (43%) and idiopathic disease (41%); and was similar in patients initially admitted to the intensive care unit, admitted to the ward, or treated as outpatients. CONCLUSIONS: The transplantation rate in our cohort in the first year was low, with 1-year and 5-year survival rates similar to other cohorts. Our results suggest that a conservative approach to list children for transplantation early after presentation may be justifiable except for patients with prolonged intensive care unit or mechanical circulatory support.

Real-time transthoracic three dimensional echocardiography: normal reference data for left ventricular dyssynchrony in adolescents.

BACKGROUND: Since the introduction of cardiac resynchronization therapy, the assessment of left ventricular (LV) dyssynchrony has become increasingly important. Real-time three-dimensional echocardiography (RT-3DE) is one of the methods that is increasingly used. However, normal reference data in a young population are scarce. METHODS: RT-3DE was performed in 73 healthy adolescents aged 12 to 18 years. A systolic dyssynchrony index was derived from the dispersion of time to minimum regional volume for all 16 LV segments. RESULTS: Acquisition of RT-3DE data sets was feasible in 67 of 73 subjects (92%). LV end-diastolic volume and LV end-systolic volume were 99+/-25 and 41+/-15 mL, respectively. All controls had highly synchronized segmental function (systolic dyssynchrony index, 1.26+/-0.53%). Dyssynchrony was independent of age, weight, or length. Interobserver variability for the dyssynchrony index was 2+/-2 ms and 0.3+/-0.2% for the absolute and heart rate-corrected values, respectively. Intraobserver variability was 2.0+/-0.07 ms and 0.0+/-0.27%, respectively. CONCLUSIONS: RT-3DE could be performed in most healthy adolescents with good interobserver and intraobserver variability. Highly synchronized segmental function was present and was independent of age, weight, or length.