Table 1.
Baseline and demographic characteristics.
| All patients (N = 299) | Any PAH medication cohort (N = 199) | Prostacyclin‐enriched cohort (N = 100) | |
|---|---|---|---|
| Time from diagnosis to baseline, months; median (Q1, Q3) | 2.1 (0.4, 8.8) | 2.2 (0.4, 8.0) | 2.0 (0.5, 11.4) |
| Age at baseline, years; mean (SD) | 51.6 (12.3) | 51.4 (12.9) | 51.9 (11.0) |
| Female, n (%) | 198 (66.2) | 132 (66.3) | 66 (66.0) |
| Etiology, n (%) | |||
| Idiopathic | 192 (64.2) | 124 (62.3) | 68 (68.0) |
| Heritable/familial associated | 21 (7.0) | 19 (9.6) | 2 (2.0) |
| Connective tissue disease | 56 (18.7) | 41 (20.6) | 15 (15.0) |
| Congenital heart disease | 7 (2.3) | 0 | 7 (7.0) |
| Portal hypertension | 11 (3.7) | 5 (2.5) | 6 (6.0) |
| Drugs/toxin‐induced | 4 (1.3) | 3 (1.5) | 1 (1.0) |
| HIV | 7 (2.3) | 6 (3.0) | 1 (1.0) |
| Schistosomiasis | 1 (0.3) | 1 (0.5) | 0 |
| Baseline NYHA/WHO functional class, n (%) | |||
| I | 8 (2.7) | 6 (3.0) | 2 (2.0) |
| II | 97 (32.4) | 71 (35.7) | 26 (26.0) |
| III | 172 (57.5) | 107 (62.2) | 65 (65.0) |
| IV | 22 (7.4) | 15 (7.5) | 7 (7.0) |
| Baseline 6MWD, in meters, mean (SD) | 225.9 (112.7) | 235.5 (110.1) | 206.8 (116.0) |
| Baseline BNP, n (%) | 179 (59.9) | 114 (57.3) | 65 (65.0) |
| Median, ng/L (IQR) | 299 (110–492) | 330 (125–475) | 226 (72–565) |
| Baseline NT‐proBNP, n (%) | 159 (53.1) | 89 (44.7) | 70 (70.0) |
| Median, ng/L (IQR) | 837 (261–1450) | 933 (360–1342) | 559 (90–1611) |
| Ongoing therapy at baseline, n (%) | |||
| No therapy at baseline | 180 (60.2) | 121 (60.8) | 59 (59.0) |
| PDE‐5 inhibitor, soluble guanylate cyclase stimulator | 101 (33.8) | 65 (32.7) | 36 (36.0) |
| Endothelin receptor antagonist | 89 (29.8) | 58 (29.1) | 31 (31.0) |
| Non‐parenteral prostacyclin | 7 (2.3) | 3 (1.5) | 4 (4.0) |
| Parenteral prostacyclin | 11 (3.7) | 5 (2.5) | 6 (6.0) |
Note: Baseline visit was defined as the most recent assessment before latest treatment initiation within the index period (January 2014–March 2017).
The medication cohorts were defined as: “any PAH medication” = a randomly selected cohort consisting of a sample of ~200 patients with PAH meeting inclusion criteria; “prostacyclin‐enriched” = an oversample (augment) sample of ~100–125 patients with PAH meeting inclusion/exclusion criteria treated with prostacyclin‐class agent as the latest therapy initiated during the index period for ≥90 days, approximately half of which received a parenteral prostacyclin‐class agent.
Abbreviations: 6MWD, 6‐min walk distance; BNP, brain natriuretic peptide; CTEPH, chronic thromboembolic pulmonary hypertension; IQR, interquartile range; NT‐proBNP, N‐terminal pro‐brain natriuretic peptide; NYHA, New York Heart Association; PAH, pulmonary arterial hypertension; PDE, phosphodiesterase; SD, standard deviation; WHO, World Health Organization.