Congenital heart disease (CHD) remains the leading cause of death from birth defects worldwide. While survival exceeds 90% in high-income countries, many patients—particularly where surgery is limited—rely on pharmacologic management. Treatments often reflect adult heart failure regimens, targeting shared mechanisms like neurohormonal activation and cardiac remodeling.

Highlights:

• ACE inhibitors, ARBs, beta-blockers, and diuretics to manage heart failure and ventricular dysfunction
• Endothelin receptor antagonists, PDE-5 inhibitors, prostaglandins, and sGC stimulators for pulmonary arterial hypertension (PAH)
• Digoxin, antiarrhythmics, and NSAIDs as adjunctive therapies for symptoms, arrhythmias, and ductal patency
• Up to 85% of cardiovascular drugs in children are used off-label due to lack of pediatric-specific trials

What Sets This Study Apart:

This review consolidates pharmacologic strategies tailored to the complexity of CHD across age groups and comorbidities. It emphasizes treatment rationale, dosing nuances, and highlights critical evidence gaps that affect pediatric prescribing.

Limitations:

Current practice leans heavily on adult data. Clinical trials for pediatric CHD are scarce (<0.45% of NIH-funded CV trials), limiting evidence-based dosing, safety, and outcome data in children.

How do you balance limited pediatric trial data with the need for evidence-based care in CHD? What role could personalized, genomics-driven approaches play in shaping future outcomes?