End points and clinical trial design in pulmonary arterial hypertension.

Published

Journal Article (Review)

New and emerging therapies might provide benefit in patients with pulmonary arterial hypertension. Their efficacy and safety will be compared with existing combination therapies in randomized clinical trials. Appropriate end points for these trials need to be identified: these will include exercise testing, the composite end point of time to clinical worsening, and hemodynamic markers, including advanced imaging modalities and biomarkers. Quality-of-life questionnaires are useful and important secondary end points; pulmonary arterial hypertension-specific questionnaires are currently being developed. Advantages and disadvantages of various trial designs, including placebo-controlled monotherapy or add-on trials, noninferiority studies, and withdrawal trials are also discussed.

Full Text

Cited Authors

  • McLaughlin, VV; Badesch, DB; Delcroix, M; Fleming, TR; Gaine, SP; Galiè, N; Gibbs, JSR; Kim, NH; Oudiz, RJ; Peacock, A; Provencher, S; Sitbon, O; Tapson, VF; Seeger, W

Published Date

  • June 2009

Published In

Volume / Issue

  • 54 / 1 Suppl

Start / End Page

  • S97 - 107

PubMed ID

  • 19555863

Pubmed Central ID

  • 19555863

Electronic International Standard Serial Number (EISSN)

  • 1558-3597

International Standard Serial Number (ISSN)

  • 0735-1097

Digital Object Identifier (DOI)

  • 10.1016/j.jacc.2009.04.007

Language

  • eng