Published online before print November 5, 2008, doi: 10.1161/CIRCOUTCOMES.108.787176
Original Articles |
Off-Label Use of Cardiovascular Medications in Children Hospitalized With Congenital and Acquired Heart Disease
From the Division of Cardiology (S.K.P.), Department of Pediatrics, Duke University Medical Center and Duke Clinical Research Institute, Durham, NC; Child Health Corporation of America (M.H.), Shawnee Mission, Kan; Departments of Pediatrics and Medicine (A.D.S.), Carilion Medical Center, Roanoke, Va; Department of Cardiology (K.J.J.), Children’s Hospital, Boston, Mass; Divisions of Cardiology and Critical Care, Department of Pediatrics (B.S.M.), Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio; Divisions of Cardiology (M.S.C.) and Infectious Diseases (S.S.S.), Department of Pediatrics and Center for Clinical Epidemiology and Biostatistics (S.S.S.), Children’s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, Pa.
Correspondence to Sara K. Pasquali, MD, Duke Clinical Research Institute, PO Box 17969, Durham, NC 27715. E-mail sara.pasquali{at}duke.edu
Received April 24, 2008; accepted August 27, 2008.
Background— Many barriers exist to conducting pediatric cardiovascular (CV) trials, and the majority of therapies used are not evidence based. Recent legislation has aimed to stimulate pediatric research and improve drug labeling. This study describes off-label use of CV medications in children hospitalized with congenital and acquired CV disease.
Methods and Results— The 2005 Pediatric Health Information System database was queried for patients with CV International Classification of Disease, Ninth Revision codes who received CV medications. Off-label use (patient younger than US Food and Drug Administration–approved age) was described. Multivariate analysis used Poisson regression to model the number of CV drugs received off-label. There were 31 432 patients included (median age, 10.4 months; interquartile range, 30 days to 6.8 years). Congenital heart disease (67%) was the most common diagnosis. Other diagnoses included hypertension, arrhythmia, myocarditis/cardiomyopathy, pulmonary hypertension, rheumatic fever/endocarditis, and heart transplant. Seventy-eight percent received 
1 CV medication off-label, and 31% received 3 CV medications off-label. The most commonly used CV medications were furosemide, epinephrine, dopamine, lidocaine, and milrinone. The latter 3 (prescribed in 69% of patients) were used off-label in all cases. Medications studied under recent federal initiatives were prescribed in only 20% of patients, and were still used off-label 62% of the time. In multivariate analysis, heart transplant recipients (odds ratio 1.7; 95% CI 1.5 to 1.9) were most likely to receive a greater number of off-label CV medications.
Conclusions— Despite recent legislation, off-label use of CV medications in the pediatric population is common. These data highlight the need for further study to determine which treatments should be used more frequently and which are unsafe or ineffective in children hospitalized with CV disease.
Key Words: pediatrics • heart defects, congenital • pharmacology
Presented at the 2008 American College of Cardiology Scientific Sessions Young Investigators Competition.
Related Article
Circ Cardiovasc Qual Outcomes 2008 1: 69-71.
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