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

McLaughlin VV, Badesch DB, Delcroix M, Fleming TR, Gaine SP, Galiè N, et al. (2009). End points and clinical trial design in pulmonary arterial hypertension. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 54(1 supplement), s97-s107 [10.1016/j.jacc.2009.04.007].

End points and clinical trial design in pulmonary arterial hypertension

GALIE', NAZZARENO;
2009

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
2009
McLaughlin VV, Badesch DB, Delcroix M, Fleming TR, Gaine SP, Galiè N, et al. (2009). End points and clinical trial design in pulmonary arterial hypertension. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 54(1 supplement), s97-s107 [10.1016/j.jacc.2009.04.007].
McLaughlin VV; Badesch DB; Delcroix M; Fleming TR;Gaine SP; Galiè N; Gibbs JS; Kim NH;Oudiz RJ; Peacock A; Provencher S; Sitbon O; Tapson VF; Seeger W.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11585/78599
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