Background: Many treatment options are available for
pulmonary arterial hypertension (PAH), but specific recommendations for long-term treatment are unavailable. We compared prognosis in PAH patients receiving goal-oriented, sequential combination
therapy evaluated using cardiopulmonary exercise testing (CPX) parameters or conventional empiric
therapy. Methods and Results: The Goal-Oriented
Therapy Evaluated by Cardiopulmonary Exercise Testing for
Pulmonary Arterial Hypertension (GOOD EYE) study was a multicenter, retrospective/prospective study in which a total of 129 patients with newly diagnosed PAH were enrolled (goal-oriented sequential combination
therapy, n=42; conventional empiric
therapy, n=87). Patients in the goal-oriented therapy group received sequential combination
therapy, the efficacy of which was regularly evaluated using CPX parameters. Patients in the conventional empiric therapy group received conventional empiric
therapy. The primary endpoint was cardiovascular death. In the goal-oriented therapy group, plasma
brain natriuretic peptide, mean pulmonary arterial pressure, pulmonary vascular resistance, and 6-min walk test were significantly improved at 12 months compared with baseline. Survival in the goal-oriented therapy group at 1, 2, and 3 years (97.6%, 95.2%, and 86.0%, respectively) tended to be higher than that in the conventional empiric therapy group (P=0.082). Conclusions: Goal-oriented sequential combination
therapy evaluated using CPX parameters may be associated with a favorable prognosis compared with conventional empiric
therapy in patients with newly diagnosed PAH.