Why the study?
What are the dosing, safety, and efficacy considerations for sildenafil in children with pulmonary arterial hypertension?
What are the dosing, safety, and efficacy considerations for sildenafil in children with pulmonary arterial hypertension?
This review highlights the conflicting regulatory recommendations and available evidence regarding the safety and efficacy of sildenafil for pediatric pulmonary arterial hypertension.
Conflicting FDA/EMA guidance warrants caution with long-term sildenafil in pediatric PAH; leaves open definitive safety data.
Sildenafil is a phosphodiesterase type-5 inhibitor approved for treatment of pulmonary arterial hypertension (PAH) in adults. Data from pediatric trials demonstrate a similar acute safety profile to the adult population but have raised concerns regarding the safety of long-term use in children. Interpretation of these trials remains controversial with major regulatory agencies differing in their recommendations - the US Food and Drug Administration recommends against the use of sildenafil for treatment of PAH in children, while the European Medicines Agency supports its use at "low doses". Here, we review the available pediatric data regarding dosing, acute, and long-term safety and efficacy of sildenafil for the treatment of PAH in children.
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Hill et al. (2015) studied this question.
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