Mori H, Yoshikawa T, Kimura H, Ono H, Kato H, Ono Y, Nii M, Shindo T, Inuzuka R, Horigome H, Miura M, Ogawa S, Shiono J, Furutani Y, Ishido M, Nakanishi T. Circ J. 2021 Dec 24;86(1):109-115. doi: 10.1253/circj.CJ-20-1239. Epub 2021 Sep 28. PMID: 34588404
Take Home Points:
- Pediatric DCM continues to carry significant morbidity and mortality resulting in death or transplantation in about half of patients by 10 years from diagnosis. Of note, LV dysfunction was defined as a FS below 20% per the WHO criteria.
- Age at diagnosis is not a predictor of outcome in Japanese children, unlike prior reports from the US and Australia.
- The most significant risk factor for death or transplant is a FS <10% at diagnosis.
- Family history of DCM was not associated with outcome- that may be related to the specific genetic mutations found in Japan.
- This study includes patients starting in the early 90s, therefore not all patients were started on ACE inhibitors or beta blockers. The initiation of ACE inhibitors but not beta blockers seemed to be protective in this population
- Prospective multicenter studies are needed to further delineate the course of pediatric dilated cardiomyopathy in the current era.
Commentary from Dr. Anna Tsirka (Connecticut, USA), Section editor of Pediatric & Fetal Cardiology Journal Watch.
Idiopathic dilated cardiomyopathy (DCM) reportedly carries worse prognosis in children than in adults. The outcome may vary by country and health care systems.
This represents a retrospective nationwide observational study of children with DCM in Japan between January 2010 and December 2014.
18 institutions across participated. Children younger than 18 years of age were included. Secondary cardiomyopathies (neuromuscular, metabolic disease or known myocarditis) were excluded.
LV dilation was defined as LVIDd z score >2 (adjusted to BSA) by M Mode. Systolic dysfunction was defined as FS by M mode < 20%.
106 patients were included in the study.
42% were younger than 1 year of age. 9% had a first or second degree relative with DCM.
12% were asymptomatic at the time of presentation while 80% presented with symptoms of CHF, and 4% with arrhythmia or aborted sudden death.
Echocardiographic parameters at diagnosis are presented below:
Median follow up was 3.3 years.
At last follow up alive, 98% were receiving medical treatment as shown below:
During the period of the study, 15% of patients underwent heart transplantation and 29% died without transplantation.
Freedom from death or transplantation at 1,3,5,10 and 20 years from diagnosis was 76%, 66%, 61%, 54%, and 43%, respectively. Overall survival rates (with or without transplantation) were 81%, 75%, 72%, and 53%, respectively, as shown below:
On multivariate analysis, the only predictor of death or transplant was a FS at presentation < 10%. Age at diagnosis was not a risk factor.
There was no significant difference in freedom from death or transplantation between patients treated with and those without β-blocker treatment as shown below: