Off-Label Drug Use in a Single-Center Pediatric Cardiac Intensive Care Unit

Background: The frequency of off-label drug use and its association with morbidity and mortality in the cardiac intensive care unit (CICU) has not been previously studied. Methods: Patients less than 18 years of age admitted to the CICU from June to August 2008 were retrospectively identified. Patient demographics were collected for 30 days or until CICU discharge. Off-label drug use was defined as the prescription of a medication that lacked a labeled indication based on patient's age as reported in the Micromedex drug database and electronic Physician's Desk Reference. Results: Eighty-two patients were admitted to the CICU during the study period. In all, 40 (46%) patients were male; the median age was 10.6 months. Common diagnoses were left-to-right shunt lesions (20.7%) and single-ventricle lesions (20.7%), with an overall mortality of 2.4%. Of all drugs prescribed, 36% were off-label. In all, 94% of the patients received >= 1 drug off-label. The median number of drugs prescribed off-label was four. Patients receiving more than four off-label medications were younger, had longer CICU lengths of stay (median 9.5 vs 2 days, P < .001), and increased ventilator days (median two vs one day, P < .001). Conclusions: Off-label drug use in the CICU is common. Frequency of use is likely higher in patients with a higher severity of illness. Further safety, efficacy, and pharmaceutical trials are warranted to optimize the use of these drugs to improve outcomes.