Predictors of Disease Progression in Pediatric Dilated Cardiomyopathy

Abstract

Background Despite medical advances, children with dilated cardiomyopathy (DCM) remain at high risk of death or need for cardiac transplantation. We sought to identify predictors of disease progression in pediatric DCM. Methods and Results The Pediatric Heart Network evaluated chronic DCM patients with prospective echocardiographic and clinical data collection during an 18-month follow-up. Inclusion criteria were age <22 years and DCM disease duration >2 months. Patients requiring intravenous inotropic/mechanical support or listed status 1A/1B for transplant were excluded. Disease progression was defined as an increase in transplant listing status, hospitalization for heart failure, intravenous inotropes, mechanical support, or death. Predictors of disease progression were identified using Cox proportional hazards modeling and classification and regression tree analysis. Of the 127 patients, 28 (22%) had disease progression during the 18-month follow-up. Multivariable analysis identified older age at diagnosis (hazard ratio=1.14 per year; P<0.001), larger left ventricular (LV) end-diastolic M-mode dimension z-score (hazard ratio=1.49; P<0.001), and lower septal peak systolic tissue Doppler velocity z-score (hazard ratio=0.81; P=0.01) as independent predictors of disease progression. Classification and regression tree analysis stratified patients at risk of disease progression with 89% sensitivity and 94% specificity based on LV end-diastolic M-mode dimension z-score ≥7.7, LV ejection fraction <39%, LV inflow propagation velocity (color M-mode) z-score <-0.28, and age at diagnosis ≥8.5 months. Conclusions In children with chronic stable DCM, a combination of diagnosis after late infancy and echocardiographic parameters of larger LV size and systolic and diastolic function predicted disease progression.

Pediatric and adult dilated cardiomyopathy represent distinct pathological entities

“…In contrast, coronary microvascular density was indistinguishable from adult donor controls across each adult DCM age group ( Adverse remodeling, disease duration, and comorbidities. As the duration of disease significantly differs between pediatric and adult DCM, with adult patients having longer periods of disease (24)(25)(26) (Table 1), we viewed heart failure duration as a confounding variable that may potentially explain why children with DCM display less adverse remodeling compared with adult DCM patients. To account for disease duration (defined as the period of time between clinical diagnosis of heart failure and myocardial specimen collection), we divided our adult DCM cohort into three groups: disease duration less than 1 year, disease duration 1-5 years, and disease duration greater than 5 years.…”

Dilated cardiomyopathy and severe heart failure. An update for pediatricians

“…28 Another study determined that an end-diastolic LV dimension with a Z-score > 7.7 and a LV flow propagation velocity with a Z-score > -0.28 were predictors of disease progression. [27][28][29][30] Elevated biomarkers, such as atrial natriuretic peptide and BNP, are considered as poor prognostic factors, even in asymptomatic patients, and a direct correlation has been observed with the echocardiographic dimension used during the cardiological follow-up of these patients: enddiastolic LV measurement, isovolumic contraction time of the LV, Tei index, and M-mode assessment of the interventricular septum diameter in systole and diastole. 27 It has also been observed that, although less specific than DCM, an increase in uric acid and procalcitonin levels may correlate with a poor prognosis, especially among patients with pulmonary hypertension.…”

Miocardiopatía dilatada e insuficiencia cardíaca grave. Puesta al día para el médico pediatra

“…28 Otro estudio determinó que una medición de dimensión fin de diástole del VI con puntaje z > 7,7 y velocidad de propagación de flujo VI con puntaje z > -0,28 eran predictores de progresión de la enfermedad. [27][28][29][30] La elevación de biomarcadores, como el péptido natriurético atrial y el PNC, se considera un factor de mal pronóstico en pacientes incluso asintomáticos, y se encuentra una correlación directa con la dimensión ecográfica utilizada en el seguimiento cardiológico de estos pacientes: medición del VI en fin de diástole, tiempo de contracción isovolumétrica izquierda, índice de Tei y evaluación en modo M del diámetro del septum interventricular en sístole y diástole. 27 También se ha encontrado, aunque menos específico que la MCD, que el aumento del ácido úrico y de procalcitonina puede correlacionarse con un mal pronóstico, especialmente, en los que presentan hipertensión pulmonar.…”