Author:

Keywords:

Pulmonary Arterial Hypertension, clinical trial, Science & Technology, Life Sciences & Biomedicine, Cardiac & Cardiovascular Systems, Cardiovascular System & Cardiology, clinical trial design, PAH, end points, BRAIN NATRIURETIC PEPTIDE, ENDOTHELIN-RECEPTOR ANTAGONIST, VENTRICULAR PRESSURE-OVERLOAD, CONNECTIVE-TISSUE DISEASE, PLACEBO-CONTROLLED TRIAL, AIR-FLOW LIMITATION, SERUM URIC-ACID, QUALITY-OF-LIFE, DOUBLE-BLIND, 6-MINUTE WALK, Clinical Trials as Topic, Endpoint Determination, Exercise Test, Hemodynamics, Humans, Hypertension, Pulmonary, Quality of Life, Randomized Controlled Trials as Topic, Research Design, Respiratory Function Tests, Treatment Outcome, 1102 Cardiorespiratory Medicine and Haematology, 1117 Public Health and Health Services, Cardiovascular System & Hematology, 3201 Cardiovascular medicine and haematology

Abstract:

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.