Sacubitril-valsartan for previously untreated chronic heart failure with reduced ejection fraction in children

Sacubitril-valsartan is currently in clinical development for the treatment of paediatric chronic
heart failure (HF) with reduced ejection fraction. HF is a clinical syndrome of symptoms and signs
that suggest the efficiency of the heart to pump blood around the body is impaired. The classical
presenting symptoms of HF in young children are feeding difficulties, growth failure, irritability,
and respiratory distress, while in older patients fatigue and exercise intolerance are more
prevalent. More than half of people with HF have reduced ejection fraction (HFrEF) which means
the heart muscle does not contract effectively and therefore less oxygen-rich blood is pumped
out to the body. Most guidelines recommending drug therapy for paediatric HF are extrapolated
from studies in adults. There remains a large unmet need for new therapies in the treatment of
paediatric HFrEF.