Table 6

Characteristics of RCTs with PAH drugs testing upfront combination therapy

Drug(s) testedStudyPatients numberDuration (weeks)Background TherapyPrimary end pointMain results
Epoprostenol vs epoprostenol+bosentanBREATHE-2813312NoPVRPVR not improved
6MWD not improved
Bosentan, sildenafil, intravenous epoprostenolSitbon821916No6MWD6MWD improved
Ambrisentan or tadalafil vs ambrisentan+tadalafilAMBITION8350078NoTTCWTTCW improved
6MWD improved
(SSc only)
RV mass (MRI)
PVR improved
RV mass improved
  • Modified from Galiè N et al.12

  • Note that most of the listed RCT data were derived from studies in adults with PAH. Healthcare providers must obtain valid information on the approval of any of the listed medications for use in paediatric PAH in the according country.

  • 6MWD, 6 min walk distance; PAH, pulmonary arterial hypertension; PVR, pulmonary vascular resistance; RCT, randomised controlled trial; RV, right ventricle; TTCW, time to clinical worsening; SSc, systemic sclerosis.