Natural history children
of dilated
cardiomyopathy
in
To assess the natural history and potential risk factors in childhood dilated cardiomyopathy, we investigated 25 patients (ages 9.6 ? 4.4 years) who presented after they were 2 years old. All patients had symptoms of congestive heart failure and reduced contractility with a dilated left ventricle at presentation. Two factors at presentation were significantly different between patients who died less than 1 year after the presentation (n = 14) and those who survived for more than 1 year (n = 9); cardiothoracic ratio (65.1% + 6.8% vs 57.1% * 6.1%, p < 0.01) and left ventricular ejection fraction (31.3% + 7.0% vs 40.0% -t 6.2%, p < 0.05). Irrespective of intensive medical therapy, dilated cardiomyopathy in children had a poor prognosis; the actuarial survival rate was 41% at 1 year and 20% at 3 years. Other forms of therapy should be considered in the early stages of dilated cardiomyopathy in this high-risk group. (AM HEART J lgg1;121:1502.)
Teiji Akagi, MD,” Lee N. Benson, MD, FRCP(Qa Nancy E. Lightfoot, PhD,” Kerrison Chin, BSc> Greg Wilson, MD,b and Robert M. Freedom, MD, FRCP(C).” Toronto,
Ontario,
Canada
Dilated cardiomyopathy, which is characterized by a dilated poorly functioning heart, is of undetermined causes.’ There are relatively few reports on the natural history of this lesion in the pediatric population because of the small patient population and the unique difficulties in distinguishing among other forms of myocardial disease that are associated with ventricular dilatation, such as endocardial fibroelastosis or myocarditis. 2-5 Additionally, there may be a unique age influence on this natural history. In this regard, we have previously reported our experience with patients with so-called endocardial fibroelastosis who presented within the first 2 years of life4 and revealed a different survival pattern as compared with those who presented after they were 2 years old.6 The purpose of this retrospective study was to assess the natural course of dilated cardiomyopathy (nonendocardial fibroelastosis) in that group of patients who were over 2 years of age at presentation and to attempt to define predictive factors that could define a subgroup with a poor prognosis. From the BDepartment of Pediatrics, Division of Cardiology, the Variety Club Catheterization Laboratories, and the bDepartment of Pathology, The Hospital for Sick Children, Toronto, Ontario, Canada. Received
for publication
May
29, 1990;
Reprint requests: Lee N. Benson, tal for Sick Children. 555 University 1X8. 4/l/27808
1502
accepted
Oct. 10, 1990.
MD, Division of Cardiology, Ave., Toronto, Ontario,
The HospiCanada, M5G
METHODS
The computerized cardiac data base of the Hospital for Sick Children, Toronto, was reviewed for patients with a diagnosis of cardiomyopathy who had presented after they were 2 years old. Four hundred and forty-seven patients were identified in the period between 1970 and 1989, and their charts were reviewed. Criteria for inclusion in the study and the diagnosis of dilated cardiomyopathy included the following: (1) clinical signs of congestive heart failure; (2) a dilated and poorly contractile left ventricle as determined by echocardiogram or angiogram; (3) absence of congenital cardiac anomalies, valvular heart disease, or chronic arrhythmias; and (4) absence of other forms of cardiomyopathy (i.e., restrictive, hypertrophic, or obstructive), acute myocarditis, or specific heart-muscle diseases. Twenty-five patients were identified (5.6%), and in 17 (68%) the diagnosis was confirmed histologically (in 13 by autopsy, in 3 by left ventricular endomyocardial biopsy, and in 1 by both procedures). Clinical and hemodynamic assessment. Clinical findings at initial evaluation were reviewed from the cardiac record and included the following variables: (1) age at onset; (2) sex; (3) body weight; (4) family history; (5) cardiothoracic ratio in chest roentgenogram; (6) systolic and diastolic blood pressure; (7) electrocardiographic findings, including ST-T wave changes, frontal QRS axis, ventricular or atria1 hypertrophy, and the presence of arrhythmias; (8) left ventricular ejection fraction as determined by echocardiogram; (9) treatment; and (10) outcome. Cardiac catheterization data were available for 16 patients (64%); catheterization was performed within 7 months of presentation (average 1.5 -+ 1.7 months).
“c.l”rm Number
121 5
Prognostic risk factors. To determine factors that were predictive of death within 1 year of diagnosis,clinical and hemodynamic findings were comparedbetween the group of 14 patients who died and the group of 9 patients who survived beyond 1 year after presentation. Statistical analysis. Cumulative actual survival wasdetermined with the Kaplan-Meier method? The significanceof the clinical and hemodynamic findings were evaluated by the two-tailed Student’s t test and chi square analysis. A value of p < 0.05 was consideredstatistically significant. RESULTS Clinical
findings. There were 15 (60%) male and 10 (40%) female patients. The mean age at diagnosis was 9.6 ? 4.4 years (range 3 to 18.1 years), and the follow-up period was 15.6 * 23.4 months (range 1 day to 7.8 days). Body weight averaged 31.9 + 13.3 kg. Two patients (8%) were siblings, and both died
within 1 year.
At the time of initial presentation, all patients had symptoms of congestive heart failure. As determined from chest roentgenograms, the cardiothoracic ratio ranged from 50% to 80% (62.4% +- 7.4%), and in 22 (88 % ) patients it was >55 % . Systolic blood pressure averaged 91.0 & 13.3 mm Hg, and diastolic pressure averaged 61.3 + 10.0 mm Hg. All patients had abnormal EGG findings at presentation. ST-T wave abnormalities were noted in 23 patients (92 % ): left ventricular hypertrophy with ST-T wave flattening or inversion (leads Vg and Vs) in nine, right ventricular hypertrophy with ST-T wave elevation in the right precordial leads in four, combined ventricular hypertrophy with ST-T wave inversion (leads V5 and Vs) in one, low QRS voltages with ST-T wave inversion (leads Vg and Vs) in four, and ST-T wave flattening or inversion in the left precordial leads in five patients. Sixteen patients (64 % ) had left atria1 or combined atria1 enlargement. The frontal QRS axis was normal (0 to +lOO degrees) in 13 patients (52% ), rightward (>lOO degrees) in six (24 % ), and leftward (40 mm Hg in 6 of 16 patients (38% ). All died during the follow-up period; the mean survival period was 27.0 * 34.2 months. Mean right atria1 pressures were elevated >lO mm Hg in 7 patients (44%), left ventricular end-diastolic pressures were >15 mm Hg in 10 (63% ), and the cardiac indexes were
of dilated
cardiomyopathy
in
To assess the natural history and potential risk factors in childhood dilated cardiomyopathy, we investigated 25 patients (ages 9.6 ? 4.4 years) who presented after they were 2 years old. All patients had symptoms of congestive heart failure and reduced contractility with a dilated left ventricle at presentation. Two factors at presentation were significantly different between patients who died less than 1 year after the presentation (n = 14) and those who survived for more than 1 year (n = 9); cardiothoracic ratio (65.1% + 6.8% vs 57.1% * 6.1%, p < 0.01) and left ventricular ejection fraction (31.3% + 7.0% vs 40.0% -t 6.2%, p < 0.05). Irrespective of intensive medical therapy, dilated cardiomyopathy in children had a poor prognosis; the actuarial survival rate was 41% at 1 year and 20% at 3 years. Other forms of therapy should be considered in the early stages of dilated cardiomyopathy in this high-risk group. (AM HEART J lgg1;121:1502.)
Teiji Akagi, MD,” Lee N. Benson, MD, FRCP(Qa Nancy E. Lightfoot, PhD,” Kerrison Chin, BSc> Greg Wilson, MD,b and Robert M. Freedom, MD, FRCP(C).” Toronto,
Ontario,
Canada
Dilated cardiomyopathy, which is characterized by a dilated poorly functioning heart, is of undetermined causes.’ There are relatively few reports on the natural history of this lesion in the pediatric population because of the small patient population and the unique difficulties in distinguishing among other forms of myocardial disease that are associated with ventricular dilatation, such as endocardial fibroelastosis or myocarditis. 2-5 Additionally, there may be a unique age influence on this natural history. In this regard, we have previously reported our experience with patients with so-called endocardial fibroelastosis who presented within the first 2 years of life4 and revealed a different survival pattern as compared with those who presented after they were 2 years old.6 The purpose of this retrospective study was to assess the natural course of dilated cardiomyopathy (nonendocardial fibroelastosis) in that group of patients who were over 2 years of age at presentation and to attempt to define predictive factors that could define a subgroup with a poor prognosis. From the BDepartment of Pediatrics, Division of Cardiology, the Variety Club Catheterization Laboratories, and the bDepartment of Pathology, The Hospital for Sick Children, Toronto, Ontario, Canada. Received
for publication
May
29, 1990;
Reprint requests: Lee N. Benson, tal for Sick Children. 555 University 1X8. 4/l/27808
1502
accepted
Oct. 10, 1990.
MD, Division of Cardiology, Ave., Toronto, Ontario,
The HospiCanada, M5G
METHODS
The computerized cardiac data base of the Hospital for Sick Children, Toronto, was reviewed for patients with a diagnosis of cardiomyopathy who had presented after they were 2 years old. Four hundred and forty-seven patients were identified in the period between 1970 and 1989, and their charts were reviewed. Criteria for inclusion in the study and the diagnosis of dilated cardiomyopathy included the following: (1) clinical signs of congestive heart failure; (2) a dilated and poorly contractile left ventricle as determined by echocardiogram or angiogram; (3) absence of congenital cardiac anomalies, valvular heart disease, or chronic arrhythmias; and (4) absence of other forms of cardiomyopathy (i.e., restrictive, hypertrophic, or obstructive), acute myocarditis, or specific heart-muscle diseases. Twenty-five patients were identified (5.6%), and in 17 (68%) the diagnosis was confirmed histologically (in 13 by autopsy, in 3 by left ventricular endomyocardial biopsy, and in 1 by both procedures). Clinical and hemodynamic assessment. Clinical findings at initial evaluation were reviewed from the cardiac record and included the following variables: (1) age at onset; (2) sex; (3) body weight; (4) family history; (5) cardiothoracic ratio in chest roentgenogram; (6) systolic and diastolic blood pressure; (7) electrocardiographic findings, including ST-T wave changes, frontal QRS axis, ventricular or atria1 hypertrophy, and the presence of arrhythmias; (8) left ventricular ejection fraction as determined by echocardiogram; (9) treatment; and (10) outcome. Cardiac catheterization data were available for 16 patients (64%); catheterization was performed within 7 months of presentation (average 1.5 -+ 1.7 months).
“c.l”rm Number
121 5
Prognostic risk factors. To determine factors that were predictive of death within 1 year of diagnosis,clinical and hemodynamic findings were comparedbetween the group of 14 patients who died and the group of 9 patients who survived beyond 1 year after presentation. Statistical analysis. Cumulative actual survival wasdetermined with the Kaplan-Meier method? The significanceof the clinical and hemodynamic findings were evaluated by the two-tailed Student’s t test and chi square analysis. A value of p < 0.05 was consideredstatistically significant. RESULTS Clinical
findings. There were 15 (60%) male and 10 (40%) female patients. The mean age at diagnosis was 9.6 ? 4.4 years (range 3 to 18.1 years), and the follow-up period was 15.6 * 23.4 months (range 1 day to 7.8 days). Body weight averaged 31.9 + 13.3 kg. Two patients (8%) were siblings, and both died
within 1 year.
At the time of initial presentation, all patients had symptoms of congestive heart failure. As determined from chest roentgenograms, the cardiothoracic ratio ranged from 50% to 80% (62.4% +- 7.4%), and in 22 (88 % ) patients it was >55 % . Systolic blood pressure averaged 91.0 & 13.3 mm Hg, and diastolic pressure averaged 61.3 + 10.0 mm Hg. All patients had abnormal EGG findings at presentation. ST-T wave abnormalities were noted in 23 patients (92 % ): left ventricular hypertrophy with ST-T wave flattening or inversion (leads Vg and Vs) in nine, right ventricular hypertrophy with ST-T wave elevation in the right precordial leads in four, combined ventricular hypertrophy with ST-T wave inversion (leads V5 and Vs) in one, low QRS voltages with ST-T wave inversion (leads Vg and Vs) in four, and ST-T wave flattening or inversion in the left precordial leads in five patients. Sixteen patients (64 % ) had left atria1 or combined atria1 enlargement. The frontal QRS axis was normal (0 to +lOO degrees) in 13 patients (52% ), rightward (>lOO degrees) in six (24 % ), and leftward (40 mm Hg in 6 of 16 patients (38% ). All died during the follow-up period; the mean survival period was 27.0 * 34.2 months. Mean right atria1 pressures were elevated >lO mm Hg in 7 patients (44%), left ventricular end-diastolic pressures were >15 mm Hg in 10 (63% ), and the cardiac indexes were
