Table 1.
Summary of baseline demographics and characteristics by dosing regimen and age group
| 2 mg kg –1 b.i.d. | 2 mg kg –1 t.i.d. | |||||
|---|---|---|---|---|---|---|
| <2 years n = 10 | ≥2 years n = 23 | Overall n = 33 | <2 years n = 11 | ≥2 years n = 20 | Overall n = 31 | |
| Gender, n (%) | ||||||
| Males | 1 (10.0) | 14 (60.9) | 15 (45.5) | 10 (90.9) | 11 (55.0) | 21 (67.7) |
| Females | 9 (90.0) | 9 (39.1) | 18 (54.5) | 1 (9.1) | 9 (45.0) | 10 (32.3) |
| Age (years), mean ± SD | 1.3 ± 0.50 | 5.9 ± 3.07 | 4.5 ± 3.35 | 1.1 ± 0.51 | 7.5 ± 2.74 | 5.2 ± 3.81 |
| Baseline PAH‐specific treatment, (consolidated stratification factor) a n (%) | ||||||
| Treatment‐naïve | 4 (40.0) | 9 (39.1) | 13 (39.4) | 3 (27.3) | 7 (35.0) | 10 (32.3) |
| Bosentan | 3 (30.0) | 7 (30.4) | 10 (30.3) | 3 (27.3) | 5 (25.0) | 8 (25.8) |
| Prostanoid | – | – | – | – | 1 (5.0) | 1 (3.2) |
| PDE‐5 inhibitor | 3 (30.0) | 7 (30.4) | 10 (30.3) | 5 (45.5) | 7 (35.0) | 12 (38.7) |
| Aetiology for PAH, n (%) b | ||||||
| IPAH | 3 (30.0) | 11 (47.8) | 14 (42.4) | 5 (45.5) | 10 (52.6) | 15 (50.0) |
| HPAH | 1 (10.0) | 1 (4.3) | 2 (6.1) | – | – | – |
| APAH c | 1 (10.0) | 10 (43.5) | 11 (33.3) | 5 (45.5) | 8 (42.1) | 13 (43.3) |
| PAH–CHD associated with systemic‐to‐pulmonary shunts or Eisenmenger syndrome | 5 (50.0) | 1 (4.3) | 6 (18.2) | 1 (9.1) | 1 (5.3) | 2 (6.7) |
| Time from first observed/assumed PAH symptoms d (days e ), mean ± SD | 320.0 ± 218.98 | 796.4 ± 902.59 | 601.5 ± 735.71 | 283.0 ± 200.12 | 1058.5 ± 1053.42 | 800 ± 933.44 |
| WHO FC, n (%) | ||||||
| I | 2 (20.0) | 6 (26.1) | 8 (24.2) | 3 (27.3) | 5 (25.0) | 8 (25.8) |
| II | 3 (30.0) | 10 (43.5) | 13 (39.4) | 4 (36.4) | 13 (65.0) | 17 (54.8) |
| III | 5 (50.0) | 7 (30.4) | 12 (36.4) | 4 (36.4) | 2 (10.0) | 6 (19.4) |
In the case of a combination of PAH‐specific medications, the following hierarchy was applied: bosentan > prostanoid > PDE‐5 inhibitor
One patient from the ≥2‐years t.i.d. dosing group had pulmonary hypertension associated with a congenital diaphragmatic hernia (nontargeted aetiology), which was clarified after randomization.
Persisting after complete repair of a congenital heart defect (PAH had to be persistent for at least 6 months after surgery)
Time from PAH symptoms excludes patients with an APAH aetiology
Calculated in relation to the date of screening
All‐randomized set. APAH, associated PAH; b.i.d., twice daily; HPAH, heritable PAH; IPAH, idiopathic PAH; PAH, pulmonary arterial hypertension; PAH–CHD, PAH with congenital heart disease; PDE‐5, phosphodiesterase‐type 5; SD, standard deviation; t.i.d., three times daily; WHO FC, World Health Organization functional class