A new study reports that improvements in multicomponent endpoints and risk stratification tools based on functional class (FC), 6-min walking distance (6MWD), and N-terminal fragment of pro-brain natriuretic peptide (NT-proBNP) were associated with improved survival in pulmonary arterial hypertension (PAH).

The study, published in the Journal of Heart and Lung Transplantation, also notes that individual improvements in 6MWD and NT-proBNP were of limited prognostic value.

“We assessed the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA) database to analyze the prognostic value of improvements in a variety of outcome measures including multicomponent improvement endpoints and established risk stratification tools,” the authors explained. “Our data suggest that improvements in multicomponent endpoints and established risk stratification models predict survival.”


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The prognostic value of sole endpoint measures in PAH has been studied for some time. In general, individual improvements in FC have been shown to be associated with better survival, but improvements in NT-proBNP and 6MWD have demonstrated little to no predictive value. Thus, the predictive ability of multicomponent endpoint improvements has recently begun to be investigated.

In this study, the research team analyzed data on 596 patients with newly diagnosed PAH at baseline and at the first follow-up, which occurred 3-12 months after the initiation of PAH treatment. The analysis revealed that sole improvements in FC and 6MWD had no predictive value, and individual improvements in NT-proBNP only predicted improved survival with relative declines ≥35%.

However, multicomponent endpoint improvements and risk stratification tools based on FC, 6MWD, and NT-proBNP were associated with improved survival. The authors recommend investigating these tools’ acceptability as surrogate endpoints in future clinical trials in PAH.

Reference

Hoeper M, Pausch C, Olsson K, et al. Prognostic value of improvement endpoints in pulmonary arterial hypertension trials: a COMPERA analysis. J Heart Lung Transplant. Published online March 22, 2022. doi:10.1016/j.healun.2022.03.011