Outcomes and risk assessment in pulmonary veno-occlusive disease.

Pulmonary veno-occlusive disease (PVOD) is a rare and severe subtype of pulmonary arterial hypertension (PAH). Although European Society of Cardiology/European Respiratory Society (ESC/ERS) guidelines advise assessing PAH severity at baseline and during follow-up, no existing risk assessment methods have been validated for PVOD. This study aimed to identify prognostic factors, examine the impact of treatment strategies and evaluate risk assessment methods for PVOD patients. The study analysed all incident PVOD patients included in the French Pulmonary Hypertension Registry between 2006 and 2021. Survival was assessed based on initial treatment strategy and risk status and compared to a matched (age, sex, pulmonary vascular resistance) PAH group. Six risk assessment methods (number of four low-risk and three noninvasive low-risk variables, ESC/ERS guidelines three-strata and four-strata models, REVEAL 2.0 and Lite 2) were applied at baseline and early follow-up, and their accuracy was compared using Harrell's c-statistic. Among the 327 included PVOD patients, survival rates at 1, 3 and 5 years were 86%, 50% and 27%, respectively. Multivariate analysis showed that only 6-min walk distance was associated with survival, with no significant difference based on initial treatment strategy. All six risk assessment methods could discriminate mortality risk, and the ESC/ERS four-strata model was the most accurate at both baseline and follow-up (C-index 0.64 and 0.74). PVOD survival rates were consistently lower than PAH when comparing baseline risk status using the ESC/ERS four-strata model. PVOD is associated with poor outcomes, and initial treatment strategies do not significantly affect survival. Risk assessment methods can be useful in predicting survival for PVOD patients.

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Conflict of interest: A. Boucly reports grants or contracts from Acceleron, Janssen and MSD, outside the submitted work; payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Janssen, Merck, AOP Orphan and Ferrer, outside the submitted work; support for attending meetings and/or travel from Janssen and MSD, outside the submitted work. Conflict of interest: X. Jaïs reports grants or contracts from Acceleron, Janssen, MSD and Bayer HealthCare, outside the submitted work; payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Janssen, and MSD, outside the submitted work. Conflict of interest: M. Jevnikar reports payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Janssen, outside the submitted work. Conflict of interest: A. Bourdin reports grants or contracts from AstraZeneca and Boehringer Ingelheim, outside the submitted work; consulting fees from AstraZeneca, GSK, Novartis, Sanofi Regeneron, Boehringer Ingelheim and Chiesi, outside the submitted work; payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from AstraZeneca, GSK, Novartis, Sanofi Regeneron, Boeringher Ingelheim and Chiesi, outside the submitted work; support for attending meetings and/or travel from AstraZeneca, GSK, Novartis, Sanofi Regeneron, Boehringer Ingelheim and Chiesi, outside the submitted work; and participation on a data safety monitoring or advisory board for AB Science, outside the submitted work. Conflict of interest: A. Chaouat reports payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from GSK, MSD and Chiesi, outside the submitted work; and support for attending meetings and/or travel from Janssen and AstraZeneca, outside the submitted work. Conflict of interest: V. Cottin reports consulting fees from Ferrer/United Therapeutics, outside the submitted work; payment for lectures and consulting from Ferrer/United Therapeutics, outside the submitted work; participation on data and safety monitoring board for Galapagos, Galecto and GSK, outside the submitted work; and adjudication committee for Fibrogen, outside the submitted work. Conflict of interest: L. Bertoletti reports grant to the institution for research studies from MSD and Bayer, outside the submitted work; consulting fees from MSD, outside the submitted work; payments for honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from MSD, BMS/Pfizer, LEO-Pharma and Viatris, outside the submitted work; support for attending meetings and/or travel from Johnson and Johnson, BMS/Pfizer, and LEO-Pharma, outside the submitted work; participation on a data safety monitoring or advisory board for Bayer, outside the submitted work; receipt of medical writing services from BMS/Pfizer, outside the submitted work. Conflict of interest: L. Savale reports payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Janssen, outside the submitted work; support for attending meetings and/or travel from Merck, outside the submitted work; and participation on a data safety monitoring or advisory board for Janssen, outside the submitted work. Conflict of interest: M. Humbert reports grants or contracts from Acceleron, AOP Orphan, Janssen, Merck and Shou Ti, outside the submitted work; consulting fees from Acceleron, Aerovate, Altavant, AOP Orphan, Bayer, Chiesi, Ferrer, Janssen, Merck, MorphogenIX, Shou Ti, Tiakis and United Therapeutics, outside the submitted work; payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Janssen and Merck, outside the submitted work; and participation on a data safety monitoring or advisory board for Acceleron, Altavant, Janssen, Merck, and United Therapeutics, outside the submitted work. Conflict of interest: O. Sitbon reports grants or contracts from Acceleron (now MSD), AOP Orphan, Janssen (formerly Actelion) and MSD, outside the submitted work; consulting fees from Acceleron (now MSD), Altavant (now Enzyvant), AOP Orphan, Ferrer, Gossamer Bio, Janssen (formerly Actelion) and MSD, outside the submitted work; honoraria for speaking at conferences from AOP Orphan, Janssen (formerly Actelion), Ferrer and MSD, outside the submitted work; and honoraria received for Trial Steering Committee membership (topic: pulmonary hypertension) from Altavant (now Enzyvant), Gossamer Bio and Janssen (formerly Actelion), outside the submitted work. Conflict of interest: D. Montani reports grants or contracts from Acceleron, Janssen and Merck MSD, outside the submitted work; consulting fees from Acceleron, Janssen, Merck MSD and Ferrer, outside the submitted work; and payment or honoraria for speakers’ bureaus from Bayer, Janssen, Boehringer, Chiesi, GSK, Ferrer and Merck MSD, outside the submitted work.