Table 3

Characteristics of RCTs with PAH drugs interfering with the endothelin pathway (endothelin receptors antagonists)

Drug(s) testedStudyPatients numberDuration (weeks)Background TherapyPrimary end pointMain results
AmbrisentanARIES-13420212No6MWD6MWD improved
TTCW not improved
ARIES-23419212No6MWD6MWD improved
TTCW improved
BosentanStudy-351353212No6MWD6MWD improved
TTCW improved
BREATHE-13621316No6MWD6MWD improved
TTCW improved
Sildenafil (16%)
PVR improved
TTCW improved
6MWD not improved
BREATHE-5295412NoSaO2, PVRPVR improved
6MWD improved
COMPASS-23833499SildenafilTTCWTTCW not improved
6MWD improved
NT-proBNP improved
MacitentanSERAPHIN33742115No or
sildenafil or
inhalative iloprost
TTCWTTCW improved in
monotherapy and in combination
  • 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; SaO2, finger oxygen saturation; TTCW, time to clinical worsening.