Off-label use of cardiovascular (CV) drugs in home therapy is common for children with congenital or acquired heart disease, according to a study published in The American Journal of Cardiology.

Researchers reviewed the records of patients (N=325) treated at the outpatient pediatric cardiology clinic of the Giovanni XXIII Pediatric Hospital in Italy in 2019 for home therapies used for cardiovascular disease (CVD). Medication, disease, and outcomes were assessed for safety and efficacy.

Patients had a median age of 4.0 years (IQR, 0.8-11.0), 55.1% were male, 67.1% had congenital heart disease, and 8.7% cardiomyopathy.


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Patients were receiving greater than or equal to 1 (88.3%), greater than or equal to 2 (34.7%), and greater than or equal to 3 (6.7%) off-label medications. The drugs included angiotensin converting enzyme inhibitors or angiotensin-receptor blockers (33.5%), diuretics (32.5%), beta-blockers (29.2%), anti-arrhythmic agents (21.8%), antiplatelets (19.1%), non-CV medications (6.1%), pulmonary vasodilators (4.0%), other anti-hypertensive agents (1.2%), and anticoagulants (0.9%). All patients receiving angiotensin converting enzyme inhibitors or angiotensin-receptor blockers, anti-arrhythmic agents, beta-blockers, other anti-hypertensive agents, and pulmonary vasodilators were receiving off-label medications. No patients who took anticoagulants used off-label medications.

In general, patients with congenital heart disease tended to be prescribed more off-label medications than patients without congenital heart disease.

A total of 8 patients discontinued medication use due to adverse events, 2 of whom experienced life-threatening events. All 8 patients with adverse event-related discontinuation used off-label medications.

Stratified by CVD categories (heart failure [HF], arrhythmias, arterial hypertension, sub-pulmonary stenosis, pulmonary hypertension, other), all groups differed significantly for characteristics, number of medications, safety, and efficacy (all P ≤.01).

Predictors of using greater than or equal to 2 off-label medications included single-ventricle physiology (adjusted odds ratio [aOR], 8.4; 95% CI, 2.25-54.4; P =.006) and HF (aOR, 2.0; 95% CI, 1.1-3.6; P =.01). Decreased risk for using greater than or equal to 2 off-label medications was observed among patients with cyanotic congenital heart disease (aOR, 0.3; 95% CI, 0.1-0.8; P =.01).

This study was limited by its follow-up time, in which it remains unclear what the long-term outcomes are for pediatric patients using multiple off-label medications.

The study data indicate that using off-label mediations for the treatment of CV conditions was common among the pediatric population. “This study highlights the need for larger safety and efficacy trials in this specific cohort of pediatric patients,” the study authors noted.

Reference

Meliota G, Lombardi M, Benevento M, et al. Off-label use of cardiovascular drugs in the home therapy of children with congenital or acquired heart disease. Am J Cardiol.  Published online December 21, 2021. doi:10.1016/j.amjcard.2021.11.029