Real-world observational study on pulmonary arterial hypertension: Italian cohort treated with macitentan and/or selexipag as a part of a combination treatment (INSPECTIO).
Pulmonary arterial hypertension (PAH) is a progressive disease associated with significant morbidity and mortality. Combination therapy targeting multiple pathways has been shown to improve clinical outcomes.
The INSPECTIO study was a prospective, multicenter, observational study conducted across 29 Italian centers specializing in PAH management. The study sought to explore the impact of combination therapy on disease progression and quality of life, by assessing non-invasive risk parameters, echocardiographic and hemodynamic conditions, and patient-reported outcomes (PROs). Patients with PAH at low or intermediate mortality risk, treated with macitentan and/or selexipag (as monotherapy or in combination), were enrolled. Data on non-invasive low-risk criteria (WHO Functional Class, 6-min walk distance [6MWD], and NT-proBNP), REVEAL risk scores, echocardiographic and hemodynamic parameters, and patient-reported outcomes (emPHasis-10) were collected at baseline and 12 months. A Narrative Medicine analysis complemented quantitative findings by exploring patients' experiences.
Among 176 patients with follow-up data, the number of non-invasive low-risk criteria increased by an average of 0.15 at 12 months (p = 0.0167). REVEAL 2.0 and REVEAL Lite 2 scores showed significant reductions (mean changes: -1.0, p = 0.0142; -0.8, p = 0.0111, respectively). Modest changes were observed in echocardiographic and hemodynamic parameters. Narrative analysis highlighted strengthened patient-provider relationships and improved coping strategies. Safety outcomes aligned with known profiles.
The INSPECTIO study supports the effectiveness of guideline-directed therapy and regular risk assessment to optimize treatment strategies in PAH. The increase in non-invasive low-risk criteria suggests a stabilization of disease over 12 months.
The INSPECTIO study was a prospective, multicenter, observational study conducted across 29 Italian centers specializing in PAH management. The study sought to explore the impact of combination therapy on disease progression and quality of life, by assessing non-invasive risk parameters, echocardiographic and hemodynamic conditions, and patient-reported outcomes (PROs). Patients with PAH at low or intermediate mortality risk, treated with macitentan and/or selexipag (as monotherapy or in combination), were enrolled. Data on non-invasive low-risk criteria (WHO Functional Class, 6-min walk distance [6MWD], and NT-proBNP), REVEAL risk scores, echocardiographic and hemodynamic parameters, and patient-reported outcomes (emPHasis-10) were collected at baseline and 12 months. A Narrative Medicine analysis complemented quantitative findings by exploring patients' experiences.
Among 176 patients with follow-up data, the number of non-invasive low-risk criteria increased by an average of 0.15 at 12 months (p = 0.0167). REVEAL 2.0 and REVEAL Lite 2 scores showed significant reductions (mean changes: -1.0, p = 0.0142; -0.8, p = 0.0111, respectively). Modest changes were observed in echocardiographic and hemodynamic parameters. Narrative analysis highlighted strengthened patient-provider relationships and improved coping strategies. Safety outcomes aligned with known profiles.
The INSPECTIO study supports the effectiveness of guideline-directed therapy and regular risk assessment to optimize treatment strategies in PAH. The increase in non-invasive low-risk criteria suggests a stabilization of disease over 12 months.
Authors
D'Alto D'Alto, Scelsi Scelsi, Giuliani Giuliani, Perna Perna, Baldi Baldi, Guerra Guerra, Di Poi Di Poi, Vicenzi Vicenzi, Badagliacca Badagliacca, Corda Corda, Airò Airò, Ferrero Ferrero, Ameri Ameri, Bux Bux, Agostoni Agostoni, D'Agostino D'Agostino, Casu Casu, Biancospino Biancospino, Uglietti Uglietti, De Santis De Santis
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