Abstract
Introduction Contemporary risk assessment tools categorise patients with pulmonary arterial hypertension (PAH) as low, intermediate, or high-risk. A minority of patients achieve low-risk status with most remaining intermediate-risk. Our aim was to validate a 4-strata risk assessment approach categorising patients as low, intermediate-low, intermediate-high, or high risk, as proposed by the COMPERA Registry investigators.
Methods We evaluated incident patients from the French PAH Registry and applied a 4-strata risk method at baseline and at first reassessment. We applied refined cut-points for 3 variables: World Health Organization functional class, 6-minute walk distance, and N-terminal pro-brain natriuretic peptide. We used Kaplan-Meier survival analyses and Cox proportional hazards regression to assess survival according to a 3-strata and 4-strata risk approach.
Results At baseline (n=2879), the 4-strata approach identified 4 distinct risk groups and performed better than a 3-strata method for predicting mortality. The 4-strata model discrimination was higher than the 3-strata method when applied during follow-up and refined risk categories among subgroups with idiopathic PAH, connective tissue disease-associated PAH, congenital heart disease, and portopulmonary hypertension. Using the 4-strata approach, 53% of patients changed risk category from baseline compared to 39% of patients when applying the 3-strata approach. Those who achieved or maintained a low-risk status had the best survival, whereas there were more nuanced differences in survival for patients who were intermediate-low and intermediate-high.
Conclusions The 4-strata risk assessment method refined risk prediction, especially within the intermediate risk category of patients, performed better at predicting survival and was more sensitive to change than the 3-strata approach.
Footnotes
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Conflict of interest: Athénaïs Boucly reports personal fees from Actelion, Bayer and Merck, outside the submitted work.
Conflict of interest: Jason Weatherald reports grants, personal fees and non-financial support from Janssen Inc., grants, personal fees and non-financial support from Actelion, personal fees and non-financial support from Bayer, personal fees from Novartis, outside the submitted work.
Conflict of interest: Laurent Savale reports personal fees from Actelion, personal fees from MSD, grants and personal fees from GSK, outside the submitted work.
Conflict of interest: Pascal de Groote reports consulting fees from Actelion, Janssen, MSD, Novartis, Servier, Boehringer Ingelheim, Abbott, Boston, Astra Zeneca, Bayer; lecture honoraria from Abbott, Vifor, MSD, Servier, Novartis, Astra Zeneca, Actelion, Janssen, Medtronic; outside the submitted work.
Conflict of interest: Vincent Cottin reports advisory board fees and non-financial support from Actelion, advisory board fees from Bayer/MSD, outside the submitted work.
Conflict of interest: Gregoire Prevot reports personal fees from Actelion, personal fees from GSK, outside the submitted work.
Conflict of interest: Ari Chaouat reports consulting fees from GSK, Actelion and Bayer, outside the submitted work.
Conflict of interest: François Picard has nothing to disclose.
Conflict of interest: Delphine Horeau-Langlard reports grants from Acceleron, outside the submitted work.
Conflict of interest: Arnaud Bourdin reports grants from Astra Zeneca and Boeringher Ingelheim; consulting fees from Astra Zeneca, GSK, Novartis, Boeringher Ingelheim, Chiesi, Sanofi Regeneron, Amgen; lecture honoraria from Astra Zeneca, GSK, Novartis, Boeringher Ingelheim, Chiesi, Sanofi Regeneron, Roche; travel support from Boeringher Ingelheim, Chiesi, Sanofi Regeneron, Astra Zeneca, GSK, Roche; participation on advisory boards at AB science, Astra Zeneca, GSK, Sanofi Regeneron, Novartis, Acceleron; and acted as investigator in clinical trials for Vertex, Abbvie, Galapagos, Fibrogen, Nuvaira, PulmonX, Gossamer, Acceleron; outside the submitted work.
Conflict of interest: Etienne-Marie Jutant has nothing to disclose.
Conflict of interest: Antoine Beurnier has nothing to disclose.
Conflict of interest: Mitja Jevnikar has nothing to disclose.
Conflict of interest: Xavier Jais reports grants from Bayer, Janssen and Merck; lecture honoraria from Janssen and Merck; outside the submitted work.
Conflict of interest: Gérald Simonneau reports grants and personal fees from Janssen (formerly Actelion), Bayer and MSD; personal fees from Acceleron, outside the submitted work.
Conflict of interest: David Montani reports grants from Acceleron, Janssen and Merck; steering committee fees from Acceleron; lecture honoraria from Bayer, Janssen and Merck; outside the submitted work.
Conflict of interest: Olivier Sitbon reports grants from Acceleron, Janssen, GSK and MSD; steering committee fees from Gossamer Bio, Janssen and MSD; lecture honoraria from AOP Orphan, Janssen, Ferrer and MSD; advisory board participation at Acceleron, Janssen and MSD; outside the submitted work.
Conflict of interest: Marc Humbert reports grants, steering committee consulting fees, and advisory board participation from Acceleron, Janssen and Merck; lecture honoraria from AOP, Janssen and Merck; steering committee participation at United Therapeutics; outside the submitted work.
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- Received September 6, 2021.
- Accepted October 27, 2021.
- Copyright ©The authors 2021. For reproduction rights and permissions contact permissions{at}ersnet.org