Similar management strategies needed for younger vs. older children with HCM
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Young children diagnosed with hypertrophic cardiomyopathy have a symptom burden and cardiac phenotype comparable with children presenting later in adolescence, suggesting similar management strategies are needed for preadolescent patients.
In a retrospective analysis of more than 1,200 children, researchers found that long-term outcomes, including mortality, did not differ by age of presentation; however, those presenting before age 12 years experienced adverse events at a younger age.
“Hypertrophic cardiomyopathy (HCM) in children should not be considered a different disease entity to that seen in adolescents and adults,” Juan Pablo Kaski, MD, FESC, FRCP, a consultant pediatric cardiologist and lead of the Centre for Inherited Cardiovascular Diseases at Great Ormond Street Hospital in London, told Healio. “The prevailing paradigm has been that HCM is a disease of adulthood, but our findings show that it can also present even in very young children and that it can be associated with adverse outcomes in a proportion of patients.”
Comparing younger vs. older children
In a retrospective study, Kaski and colleagues assessed the clinical presentation and natural history of 639 children younger than 12 years presenting with nonsyndromic HCM, using data from the International Paediatric Hypertrophic Cardiomyopathy Consortium (65.3% boys; median age, 7 years) and compared those data with those from 568 children diagnosed from age 12 to 16 years. The primary outcome was all-cause mortality or cardiac transplantation; secondary outcomes included first life-threatening arrhythmic event, defined as sudden cardiac death or an equivalent event determined by the treating cardiologist at each center.
At baseline, 53.6% of children had family histories of HCM, 20.9% had HF symptoms and 39.2% were prescribed cardiac medications. The median maximal left ventricular wall thickness z score was 8.7 and 27.2% of children had LV outflow tract obstruction.
During a median follow-up of 5.6 years, 6.6% of children died, of which 4.9% were due to sudden cardiac death; 3.3% underwent cardiac transplantation; and 10.8% had life-threatening arrhythmic events.
Compared with children presenting after age 12 years, a higher proportion of younger patients underwent LV septal myectomy (10.5% vs. 7.2%; P = .045), but fewer received primary prevention implantable cardioverter defibrillators (18.9% vs. 30.1%; P = .041).
There were no between-group differences in mortality or life-threatening arrhythmic events; however, the events occurred at a younger age in those presenting earlier, according to researchers.
“Clinical screening for familial HCM is warranted before the age of 12 years, which previous clinical guidelines had suggested,” Kaski told Healio. “Second, and perhaps most importantly, children should not be excluded from clinical trials and novel treatments for HCM. Prospective studies to stratify patients who are most at risk for early disease expression and adverse outcomes will allow us to deliver truly personalized treatment for HCM.”
Importance of early screening
In a related editorial, Joseph W. Rossano, MD, MS, FAAP, FAAC, a pediatric cardiologist, co-director of the cardiac center and chief of the division of cardiology at Children’s Hospital of Philadelphia, and Kimberly Y. Lin, MD, attending cardiologist in the cardiac center and the medical director of cardiomyopathy at Children's Hospital of Philadelphia, wrote that the data “provide compelling reasons for screening at-risk preadolescent children for HCM.”
“This study adds to a growing body of evidence suggesting that preadolescent HCM is a condition similar to that found in adolescents and adults, namely, that while the majority do well, a significant minority are at risk for events even early in life,” Rossano and Lin wrote. “In light of our growing understanding that HCM does not discriminate based on age, we must push to identify and study those affected at an early age.”
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For more information:
Juan Pablo Kaski, MD, can be reached at j.kaski@ucl.ac.uk.
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