Medical Therapy Excellent for Pediatric Heart Failure

For children with heart failure, medical therapy should be optimized first before moving on to treatment with a device or transplantation in children in whom medical therapy is not enough.

This was the conclusion of a talk given by Melanie Everitt, MD, Director Heart Transplantation, Children’s Hospital Colorado, Aurora, CO,  in a session entitled “Cardiomyopathies: Can We Manage Without Device or Transplant?” at the American College of Cardiology (ACC) meeting.

“Children with heart failure due to cardiomyopathy can have excellent long-term outcomes with medical therapy of their heart failure,” she said.

In her talk, Everitt highlighted data showing that transplant-free survival with stable heart failure (or myocardial recovery) can be achieved, sudden cardiac death is uncommon, and survival is improving in the current era.

For example, she cited data from a 2014 study of 38 children less than 3 years of age with dilated cardiomyopathy treated with beta-blockers and angiotensin-converting enzyme inhibitors which showed that 82% were alive without transplant at 1 year and 69% at 2 years. Left ventricular ejection fraction was improved in 19% to 46% of the survivors, and levels of B-type natriuretic peptide improved significantly from 3330 to 171 pg/ml (Rupp S, et al. Upgraded Heart Failure Leads to an Improved Outcome of Dilated Cardiomyopathy in Infants and Toddlers. Cardiol Young 2014; Dec 12:1-6).

Although she said that some children who do not respond well to medical therapy and need support from a ventricular assist device or heart transplant, over half of these children do well with medical therapy alone.

“This proportion may even be higher when the heart failure is managed by a pediatric heart failure expert with attention to the child’s individual response to the medications,” Everitt noted.

The expertise from a pediatric heart specialist is important, she stressed, to help determine which children will do well or not. “When children do poorly, it tends to be fairly early in the course of the disease and it is difficult from the child’s presentation to determine who will do well and who will not,” she said.

Further expertise from a heart failure specialist is also needed, Everitt said, once the child is stabilized and it has to be determined if medical therapy is working. At this point, “a partnership with the heart failure specialist and the front-line pediatrician is key for ongoing management and serial heart failure assessments.”

What remains unknown in the treatment of children with cardiomyopathies, she noted, is who will improve and which medical regimen works best.