Progress in Congenital Cardiac Care for Newborns and Infants: The Emerging Role of “Off-label” Medications

Congenital heart disease accounts for a major percentage of clinically significant birth defects and is the leading cause for infant deaths that are due to a congenital anomaly. Extraordinary advancements during the last three decades in noninvasive diagnosis, surgical repairs, cardiac catheterization interventions, and perioperative pediatric intensive care management have dramatically improved survival for children born with congenital heart disease. Advances in care have included management with Food and Drug Administration–approved medications, as well as many “off-label” medications. An off-label drug has been defined as a drug used for a patient “younger than the Food and Drug Administration (FDA)–approved age range for any indication of that drug” (Arch Pediatr Adolesc Med. 2007; 161: 282-290). This article will review selected off-label medications currently used in the pediatric congenital heart management for treatment of perioperative low cardiac output, congestive heart failure, pulmonary hypertension, sedation, anticoagulation, and pain.