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Future perspectives in pulmonary arterial hypertension

Gérald Simonneau, Marius M. Hoeper, Vallerie McLaughlin, Lewis Rubin, Nazzareno Galiè
European Respiratory Review 2016 25: 381-389; DOI: 10.1183/16000617.0084-2016
Gérald Simonneau
1Université Paris-Sud, Faculté de Médecine, Université Paris-Saclay, Le Kremlin-Bicêtre, France
2AP-HP, Service de Pneumologie, Hôpital Bicêtre, Le Kremlin-Bicêtre, France
3INSERM U-999, Hôpital Marie Lannelongue, Le Plessis-Robinson, France
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  • For correspondence: gerald.simonneau@bct.aphp.fr
Marius M. Hoeper
4Dept of Respiratory Medicine, Hannover Medical School and German Centre for Lung Research, Hannover, Germany
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Vallerie McLaughlin
5Dept of Internal Medicine, University of Michigan Health System, Ann Arbor, MI, USA
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Lewis Rubin
6Dept of Medicine, University of California, San Diego, CA, USA
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Nazzareno Galiè
7Dept of Experimental, Diagnostic and Specialty Medicine (DIMES), University of Bologna, Bologna, Italy
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Tables

  • Supplementary Materials
  • TABLE 1

    Potential pulmonary arterial hypertension (PAH) therapies currently in development

    TherapyClinical trial identifierClinical trial designPrimary end-pointsTreatment durationStatus (October 2016)
    Therapies targeting inflammation and immunity
     UbenimexNCT02664558Phase II, multicentre, randomised, double-blind, placebo-controlled trial in PAH patientsChange in PVR24 weeksRecruiting [17]
    NCT02736149Phase II, open-label, multicentre, extension study in PAH patientsFrequency of adverse events∼1 yearNot yet recruiting [18]
     RituximabNCT01086540Phase II, randomised, double-blind, placebo-controlled trial in PAH-SSc patientsChange in PVR24 weeksRecruiting [19]
     TocilizumabNCT02676947Phase II, open-label trial in PAH patientsIncidence and severity of adverse events; change in PVR6 monthsRecruiting [20]
    Therapies targeting mitochondrial dysfunction
     Bardoxolone methylNCT02036970Phase II, double-blind, randomised, interventional trial in pulmonary hypertension Group I, II or V patientsChange in 6MWD16 weeksPreliminary results published [21]
    NCT02657356Phase III, double-blind, early interventional trial in PAH-CTD patientsChange in 6MWD24 weeksRecruiting [22]
     GS-4997NCT02234141Phase II, dose-ranging, randomised, double-blind, placebo-controlled trial in PAH patientsChange in PVR24 weeksOngoing, not recruiting [23]
    Therapies targeting BMPR2 signalling
     TacrolimusNCT01647945Phase II, double-blind, randomised trial in PAH patientsFrequency of adverse events16 weeksTerminated due to limited funding/slow patient recruitment; follow-up multicentre phase IIb efficacy trial planned [24]
    Therapies targeting iron deficiency
     Ferinject (ferric carboxymaltose)NCT01447628Phase II, double-blind, randomised, interventional trial in IPAH, HPAH and anorexigen-associated PAH patientsChange in PVR and exercise capacity24 weeksRecruiting [25]
    NCT01847352Single-blind, nonrandomised, interventional, trial in healthy volunteers who met iron-deficient or iron-replete criteriaChange in PASP following i.v. iron infusion1 weekCompleted: April 2014 [26, 27]
     Ferrous sulfate (oral dietary  iron supplement)NCT01446848Interventional, open-label study in IPAH patients with iron deficiencyChange in zinc protoporphyrin level; change in serum ferritin level12 weeksCompleted: August 2014 [28]
    Pulmonary artery denervation
     Pulmonary arterial denervation procedurechiCTR-ONC-12002085Phase II, observational, unblinded, nonrandomised study in PAH and PAH-CTD patientsChange in PASP and 6MWD24 weeksCompleted: April 2014 [29]
    NCT02525926Single-blind, randomised, interventional efficacy study in PAH patientsMean pulmonary artery pressure26 weeksRecruiting [30]
    • PVR: pulmonary vascular resistance (as measured by right heart catheterisation); PAH-SSc: PAH associated with systemic sclerosis; 6MWD: 6-min walk distance; PAH-CTD: PAH associated with connective tissue disease; BMPR2: bone morphogenetic protein receptor type 2; IPAH: idiopathic PAH; HPAH: heritable PAH; PASP: pulmonary artery systolic pressure.

Supplementary Materials

  • Tables
  • Supplementary Material

    Please note: supplementary material is not edited by the Editorial Office, and is uploaded as it has been supplied by the author.

    N. Galie ERR-0084-2016_Galie

    M. Hoeper ERR-0084-2016_Hoeper

    V. McLaughlin ERR-0084-2016_McLaughlin

    L. Rubin ERR-0084-2016_Rubin

    G. Simonneau ERR-0084-2016_Simonneau

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Future perspectives in pulmonary arterial hypertension
Gérald Simonneau, Marius M. Hoeper, Vallerie McLaughlin, Lewis Rubin, Nazzareno Galiè
European Respiratory Review Dec 2016, 25 (142) 381-389; DOI: 10.1183/16000617.0084-2016

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Future perspectives in pulmonary arterial hypertension
Gérald Simonneau, Marius M. Hoeper, Vallerie McLaughlin, Lewis Rubin, Nazzareno Galiè
European Respiratory Review Dec 2016, 25 (142) 381-389; DOI: 10.1183/16000617.0084-2016
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  • Article
    • Abstract
    • Abstract
    • Introduction
    • Potential PAH therapies currently in development
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  • Pulmonary vascular disease
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