End points and clinical trial design in pulmonary arterial hypertension

J Am Coll Cardiol. 2009 Jun 30;54(1 Suppl):S97-S107. doi: 10.1016/j.jacc.2009.04.007.


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.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic*
  • Endpoint Determination*
  • Exercise Test
  • Hemodynamics
  • Humans
  • Hypertension, Pulmonary* / diagnosis
  • Hypertension, Pulmonary* / physiopathology
  • Hypertension, Pulmonary* / therapy
  • Quality of Life
  • Randomized Controlled Trials as Topic
  • Research Design
  • Respiratory Function Tests
  • Treatment Outcome