End points and clinical trial designs in pulmonary arterial hypertension: Clinical and regulatory perspectives

Marius M. Hoeper, Ronald J. Oudiz, Andrew Peacock, Victor F. Tapson, Sheila G. Haworth, Adaani E. Frost, Adam Torbicki

Research output: Contribution to journalArticlepeer-review

147 Scopus citations


To date, randomized controlled clinical trials performed in pulmonary arterial hypertension (PAH) have been relatively short-term studies involving mainly patients with advanced disease. The primary end points in these trials have addressed exercise capacity, usually by using the 6-min walk test. Although this approach is still warranted in future trials assessing new treatments, it is likely that the focus will shift toward trials of longer duration, involving patients with less advanced disease, and that different drugs and drug-combination regimens will be compared. In such trials, it is possible that a composite of markers indicating clinical deterioration (e.g., hospitalization for right heart failure, the requirement for the introduction of an alternative treatment, and predefined indicators of worsening exercise tolerance) may be more useful as primary end points. Quality of life will become a very important issue; however, appropriate quality-of-life questionnaires for PAH have yet to be developed. In addition, hemodynamics will likely remain valuable as secondary end points, but future clinical trials should include hemodynamics obtained both during exercise and at rest. Finally, cardiopulmonary exercise testing, echocardiographic studies, and biochemical parameters, such as brain natriuretic peptide or troponin T, may also prove useful as secondary end points in the future.

Original languageEnglish (US)
Pages (from-to)S48-S55
JournalJournal of the American College of Cardiology
Issue number12 SUPPL.
StatePublished - Jun 16 2004


  • BNP
  • CO
  • CPET
  • EMEA
  • European Agency for the Evaluation of Medicinal Products
  • FDA
  • Food and Drug Administration
  • LV
  • NYHA
  • New York Heart Association
  • PAH
  • brain natriuretic peptide
  • cardiac output
  • cardiopulmonary exercise testing
  • left ventricle/ventricular

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine


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