Characteristics of RCTs with PAH drugs interfering with the endothelin pathway (endothelin receptors antagonists)
| Drug(s) tested | Study | Patients number | Duration (weeks) | Background Therapy | Primary end point | Main results |
|---|---|---|---|---|---|---|
| Ambrisentan | ARIES-134 | 202 | 12 | No | 6MWD | 6MWD improved TTCW not improved |
| ARIES-234 | 192 | 12 | No | 6MWD | 6MWD improved TTCW improved | |
| Bosentan | Study-35135 | 32 | 12 | No | 6MWD | 6MWD improved TTCW improved |
| BREATHE-136 | 213 | 16 | No | 6MWD | 6MWD improved TTCW improved | |
| EARLY37 | 185 | 24 | No Sildenafil (16%) | PVR, 6MWD | PVR improved TTCW improved 6MWD not improved | |
| BREATHE-529 | 54 | 12 | No | SaO2, PVR | PVR improved 6MWD improved | |
| COMPASS-238 | 334 | 99 | Sildenafil | TTCW | TTCW not improved 6MWD improved NT-proBNP improved | |
| Macitentan | SERAPHIN33 | 742 | 115 | No or sildenafil or inhalative iloprost | TTCW | TTCW 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.