Heart of Health Policy | In March, the U.S. Food and Drug Administration (FDA) announced it is clarifying its previous recommendation related to prescribing sildenafil (Revatio) for children with pulmonary arterial hypertension (PAH). Sildenafil is FDA-approved only to treat PAH in adults, not in children; however, the FDA says health care professionals must consider whether the benefits of treatment with the drug are likely to outweigh its potential risks for each patient.

“The Adult Congenital and Pediatric Cardiology community would like to thank the FDA for issuing this important clarification related to the intent of its prior warning,” said Kathy J. Jenkins, MD, MPH, FACC, immediate past chair of the ACC’s Adult Congenital and Pediatric Cardiology Council. “Children with pulmonary hypertension and certain related conditions have limited available treatments, and sildenafil at lower doses is an important option. This clarification should remove any barriers to prescribing the drug in the right circumstances.”

FDA revised the sildenafil drug label in August 2012, adding a warning stating that “use of [sildenafil], particularly chronic use, is not recommended in children.” This recommendation was based on an observation of increasing mortality with increasing sildenafil doses in a long-term clinical trial in pediatric patients with PAH.

According to the FDA, the purpose of the recommendation was to raise awareness of clinical trial results showing a higher risk of mortality in pediatric patients taking a high dose of sildenafil when compared to pediatric patients taking a low dose. However, the agency says the recommendation was not intended to suggest that sildenafil should never be used in children.

“This issue highlights the important problem of the limited studies of drugs for rare pediatric diseases compared to adults,” Jenkins noted. “Pediatric cardiologists must extrapolate from many sources of information, as published studies are few, in order to devise the best strategies for children.”

Keywords: Health Policy, United States Food and Drug Administration, Hypertension, Pulmonary, Awareness, United States, Cardiology Magazine, ACC Publications

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