Evolution and optimization of clinical trial endpoints and design in pulmonary arterial hypertension.
biomarker
endpoint
pulmonary arterial hypertension
Journal
Pulmonary circulation
ISSN: 2045-8932
Titre abrégé: Pulm Circ
Pays: United States
ID NLM: 101557243
Informations de publication
Date de publication:
Jul 2023
Jul 2023
Historique:
received:
18
04
2023
revised:
20
07
2023
accepted:
25
07
2023
medline:
9
8
2023
pubmed:
9
8
2023
entrez:
9
8
2023
Statut:
epublish
Résumé
Selection of endpoints for clinical trials in pulmonary arterial hypertension (PAH) is challenging because of the small numbers of patients and the changing expectations of patients, clinicians, and regulators in this evolving therapy area. The most commonly used primary endpoint in PAH trials has been 6-min walk distance (6MWD), leading to the approval of several targeted therapies. However, single surrogate endpoints such as 6MWD or hemodynamic parameters may not correlate with clinical outcomes. Composite endpoints of clinical worsening have been developed to reflect patients' overall condition more accurately, although there is no standard definition of worsening. Recently there has been a shift to composite endpoints assessing clinical improvement, and risk scores developed from registry data are increasingly being used. Biomarkers are another area of interest, although brain natriuretic peptide and its
Identifiants
pubmed: 37554146
doi: 10.1002/pul2.12271
pii: PUL212271
pmc: PMC10405062
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
e12271Informations de copyright
© 2023 The Authors. Pulmonary Circulation published by John Wiley & Sons Ltd on behalf of Pulmonary Vascular Research Institute.
Déclaration de conflit d'intérêts
Marco Caccamo has received consulting and speaker fees from Bayer. Frank E. Harrell has received consulting fees from the American Statistical Association, Annexon Biosciences, the Arnold Foundation, AstraZeneca, and Johnson & Johnson/Janssen, receives textbook royalties from Springer, and received an honorarium for a Janssen Pulmonary Hypertension Key Opinion Advisory Committee in late 2021. None of the funding relates to the current work. Anna R. Hemnes has received consulting fees from Bayer, Gossamer Bio, Janssen, and United Therapeutics, has grants from the National Institutes of Health and the Cardiovascular Medical Research and Education Fund, and owns stock in Tenax Therapeutics.
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