Riociguat in patients with chronic thromboembolic pulmonary hypertension: Results from an early access study

Vallerie V. McLaughlin, Pavel Jansa, Jens E. Nielsen-Kudsk, Michael Halank, Gérald Simonneau, Ekkehard Grünig, Silvia Ulrich, Stephan Rosenkranz, Miguel A. Gómez Sánchez, Tomás Pulido, Joanna Pepke-Zaba, Joan Albert Barberá, Marius M. Hoeper, Jean Luc Vachiéry, Irene Lang, Francine Carvalho, Christian Meier, Katharina Mueller, Sylvia Nikkho, Andrea M. D'Armini

Research output: Contribution to journalArticle

Abstract

Background: Following positive results from the Phase III CHEST-1 study in patients with inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH), the Phase IIIb CTEPH early access study (EAS) was designed to assess the safety and tolerability of riociguat in real-world clinical practice, as well as to provide patients with early access to riociguat before launch. Riociguat is approved for the treatment of inoperable and persistent/recurrent CTEPH. Methods: We performed an open-label, uncontrolled, single-arm, early access study in which 300 adult patients with inoperable or persistent/recurrent CTEPH received riociguat adjusted from 1 mg three times daily (tid) to a maximum of 2.5 mg tid. Patients switching from unsatisfactory prior pulmonary arterial hypertension (PAH)-targeted therapy (n=84) underwent a washout period of at least 3 days before initiating riociguat. The primary aim was to assess the safety and tolerability of riociguat, with World Health Organization functional class and 6-min walking distance (6MWD) as exploratory efficacy endpoints. Results: In total, 262 patients (87%) completed study treatment and entered the safety follow-up (median treatment duration 47 weeks). Adverse events were reported in 273 patients (91%). The most frequently reported serious adverse events were syncope (6%), right ventricular failure (3%), and pneumonia (2%). There were five deaths, none of which was considered related to study medication. The safety and tolerability of riociguat was similar in patients switched from other PAH-targeted therapies and those who were treatment naïve. In patients with data available, mean±standard deviation 6MWD had increased by 33±42 m at Week 12 with no clinically relevant differences between the switched and treatment-naïve subgroups. Conclusions: Riociguat was well tolerated in patients with CTEPH who were treatment naïve, and in those who were switched from other PAH-targeted therapies. No new safety signals were observed. Trial registration: ClinicalTrials.org NCT01784562. Registered February 4, 2013.

Original languageEnglish
Article number216
JournalBMC Pulmonary Medicine
Volume17
Issue number1
DOIs
Publication statusPublished - Dec 28 2017

Keywords

  • Chronic thromboembolic pulmonary hypertension
  • Early access study
  • Riociguat

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Fingerprint Dive into the research topics of 'Riociguat in patients with chronic thromboembolic pulmonary hypertension: Results from an early access study'. Together they form a unique fingerprint.

  • Cite this

    McLaughlin, V. V., Jansa, P., Nielsen-Kudsk, J. E., Halank, M., Simonneau, G., Grünig, E., Ulrich, S., Rosenkranz, S., Gómez Sánchez, M. A., Pulido, T., Pepke-Zaba, J., Barberá, J. A., Hoeper, M. M., Vachiéry, J. L., Lang, I., Carvalho, F., Meier, C., Mueller, K., Nikkho, S., & D'Armini, A. M. (2017). Riociguat in patients with chronic thromboembolic pulmonary hypertension: Results from an early access study. BMC Pulmonary Medicine, 17(1), [216]. https://doi.org/10.1186/s12890-017-0563-7