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Patients’ perceptions on clinical trials outcomes in pulmonary arterial hypertension therapy
  1. Vicky Mai1,2,
  2. Camille Gosselin1,2,
  3. Élodie Tremblay1,2,
  4. Gabrielle Rompré1,2,
  5. Annie Christine Lajoie1,2,
  6. Jason Weatherald3,4,
  7. Jean-Christophe Lega5,6,7,
  8. Sébastien Bonnet1,2,8,
  9. Steeve Provencher1,2,8
  1. 1 Institut universitaire de cardiologie et de pneumologie de Québec, Quebec, Quebec, Canada
  2. 2 Pulmonary Hypertension Research Group (http://phrg.ca), Quebec, Quebec, Canada
  3. 3 Department of Medicine, Division of Respiratory Medicine, University of Calgary, Libin Cardiovascular Institute of Alberta, Calgary, Alberta, Canada
  4. 4 Department of Medicine, Division of Pulmonary Medicine, University of Alberta, Edmonton, Alberta, Canada
  5. 5 UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, Université de Lyon, Lyon, France
  6. 6 Department of Internal and Vascular Medicine, Hospices Civils de Lyon, Lyon, France
  7. 7 Groupe d’Etude Multidisciplinaire des Maladies Thrombotiques (GEMMAT), Hospices Civils de Lyon, Lyon, France
  8. 8 Department of Medicine, Université Laval, Quebec, Quebec, Canada
  1. Correspondence to Dr Steeve Provencher, Institut universitaire de cardiologie et de pneumologie de Québec, Quebec G1V 4G5, Canada; Steeve.Provencher{at}criucpq.ulaval.ca

Abstract

The value placed by patients and their caregivers on the components of composite outcomes in pulmonary arterial hypertension (PAH) remains unknown. We surveyed the importance of these outcomes from a patients’ and caregivers’ perspective, with participants (n=335, including 257 patients with PAH) rating individual components defining clinical worsening in PAH trials as of critical, major, mild-to-moderate or minor importance. Most outcomes were considered of major or mild-to-moderate importance to patients. Death was the only outcome considered of critical importance. Perceptions of clinical outcomes varied between patients and caregivers. Integrating patients’ perception in the elaboration of clinical trials is essential.

  • Primary Pulmonary Hypertension
  • Clinical Epidemiology

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Footnotes

  • Contributors VM, CG, ET, GR, ACL, JW, SB, J-CL and SP made substantial contributions to the conception or design of the work; or the acquisition, analysis or interpretation of data for the work. VM drafted the initial work, which was critically revised for important intellectual content by CG, ET, GR, ACL, JW, SB, J-CL and SP. VM, CG, ET, GR ACL, JW, SB, J-CL and SP approved the final version of the manuscript. VM, CG, ET, GR, ACL, JW, SB, J-CL and SP agree to be accountable for all aspects of the work.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests JW reports speaker’s fees from Actelion, Janssen and Bayer; grants from Ac-telion, Janssen and Merck; and travel support from Janssen and Actelion. SB reports speaker’s fees from Actelion, Janssen and Morphic; and grants from Janssen and Morphic. SP reports grants from Janssen, grants from AstraZeneca, grants from Resverlogix, outside the submitted work and speaker’s fees from Janssen. The other authors report no conflicts.

  • Provenance and peer review Not commissioned; externally peer reviewed.