Characteristics of RCTs with PAH drugs testing upfront combination therapy
| Drug(s) tested | Study | Patients number | Duration (weeks) | Background Therapy | Primary end point | Main results |
|---|---|---|---|---|---|---|
| Epoprostenol vs epoprostenol+bosentan | BREATHE-281 | 33 | 12 | No | PVR | PVR not improved 6MWD not improved |
| Bosentan, sildenafil, intravenous epoprostenol | Sitbon82 | 19 | 16 | No | 6MWD | 6MWD improved |
| Ambrisentan or tadalafil vs ambrisentan+tadalafil | AMBITION83 | 500 | 78 | No | TTCW | TTCW improved 6MWD improved |
| Ambrisentan+tadalafil | Hassoun84 (SSc only) | 24 | 36 | No | PVR 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.