Key result
Sildenafil is extensively used as an off-label treatment for pediatric pulmonary arterial hypertension, offering clinical benefits despite recent regulatory warnings regarding high-dose mortality.
Provides a review of sildenafil use in pediatric PAH, addressing the paucity of approved therapies and regulatory warnings against high-dose use.
May support monitored off-label sildenafil in pediatric PAH; leaves open optimal dosing and long-term safety from randomized trials.
Pulmonary arterial hypertension (PAH) is a life-threatening disease of varied etiologies. Although PAH has no curative treatment, a greater understanding of pathophysiology, technological advances resulting in early diagnosis, and the availability of several newer drugs have improved the outlook for patients with PAH. Sildenafil is one of the therapeutic agents used extensively in the treatment of PAH in children, as an off-label drug. In 2012, the United States Food and Drug Administration (USFDA) issued a warning regarding the of use high-dose sildenafil in children with PAH. This has led to a peculiar situation where there is a paucity of approved therapies for the management of PAH in children and the use of the most extensively used drug being discouraged by the regulator. This article provides a review of the use of sildenafil in the treatment of PAH in children.
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Dhariwal et al. (2015) conducted a review in Pediatric pulmonary arterial hypertension. Sildenafil was evaluated. Sildenafil is extensively used as an off-label treatment for pediatric pulmonary arterial hypertension, offering clinical benefits despite recent regulatory warnings regarding high-dose mortality.
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