A New START for Sildenafil in Pediatric Pulmonary Hypertension

Abstract

Pharmacotherapy for pulmonary arterial hypertension (PAH) has evolved dramatically over the past decade. One of the agents at the vanguard of this evolution was sildenafil, which was reported in 2005 to improve exercise capacity, functional status, and hemodynamics in a large randomized controlled trial (RCT) of adults with PAH (SUPER-1) 1 , and has since become a therapeutic mainstay 2 . Based in part on the promising early findings of SUPER-1, sildenafil was studied in treatment-naive children and adolescents as part of the first ever RCT for pediatric PAH, STARTS-1, which randomized patients to placebo or 1 of 3 dose-ranges of sildenafil 3 . After the 16-week evaluation, patients were allowed to remain on therapy in the extension trial, STARTS-2, which continued sildenafil in treated patients and randomized those in the placebo arm to one of the sildenafil doses. STARTS-1 enrolled 234 patients over 4.5 years at 32 centers, the vast majority of whom continued in STARTS-2.