Table 3.
Clinical Management and Outcome in the Overall Pediatric Pulmonary Hypertension Cohort and for Groups 1 and 3 Pulmonary Hypertension
| Overall (n = 1101) | Group 1 (n = 529) | Group 3 (n = 356) | P Value (Group 1 vs. 3) | |
|---|---|---|---|---|
| Clinical management | ||||
| PH therapy use at any time | 910 (82.7) | 437 (82.6) | 309 (86.8) | 0.11 |
| Initial PH therapeutic strategy | ||||
| Monotherapy | 617 (56) | 266 (50.3) | 238 (66.9) | <0.001 |
| Dual agent combination therapy | 157 (14.3) | 90 (17.0) | 39 (11.0) | 0.02 |
| Triple therapy | 46 (4.2) | 34 (6.4) | 6 (1.7) | 0.002 |
| None | 281 (25.5) | 139 (26.3) | 73 (20.5) | 0.06 |
| Initial monotherapy | ||||
| PDE5 inhibitor | 525 (47.7) | 193 (36.5) | 234 (65.7) | <0.001 |
| ERA | 64 (5.8) | 54 (10.2) | 2 (0.6) | <0.001 |
| Prostanoid | 3 (0.3) | 2 (0.4) | 0 (0) | 0.66 |
| Therapy at end of follow-up | ||||
| Monotherapy | 390 (35.4) | 168 (31.8) | 135 (37.9) | 0.07 |
| Dual agent combination therapy | 197 (17.9) | 135 (25.5) | 29 (8.1) | <0.001 |
| Triple therapy | 102 (9.3) | 83 (15.7) | 14 (3.9) | <0.001 |
| None | 412 (37.4) | 143 (27.0) | 178 (50) | <0.001 |
| Clinical outcomes | ||||
| Resolution within 5 yr of diagnosis, %* | 23.6 | 10.7 | 38.6 | — |
| Transplanted lung or heart within 5 yr of diagnosis, %* | 2.7 | 3.5 | 1.1 | — |
Definition of abbreviations: ERA = endothelin receptor antagonist; PDE5 = phosphodiesterase 5; PH = pulmonary hypertension.
*
Outcome statistics are based on the competing risks analysis. Continuous variables are presented as median [interquartile range] or mean ± standard deviation. Categorical variables are presented as n (%). P < 0.05 indicative of statistical significance.