Title |
Riociguat in patients with chronic thromboembolic pulmonary hypertension: results from an early access study
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Published in |
BMC Pulmonary Medicine, December 2017
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DOI | 10.1186/s12890-017-0563-7 |
Pubmed ID | |
Authors |
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 |
Abstract |
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. 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. 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. 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. ClinicalTrials.org NCT01784562 . Registered February 4, 2013. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Unknown | 2 | 100% |
Demographic breakdown
Type | Count | As % |
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Practitioners (doctors, other healthcare professionals) | 1 | 50% |
Members of the public | 1 | 50% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 72 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Other | 10 | 14% |
Researcher | 7 | 10% |
Student > Master | 7 | 10% |
Student > Bachelor | 5 | 7% |
Student > Ph. D. Student | 5 | 7% |
Other | 6 | 8% |
Unknown | 32 | 44% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 15 | 21% |
Pharmacology, Toxicology and Pharmaceutical Science | 7 | 10% |
Business, Management and Accounting | 3 | 4% |
Nursing and Health Professions | 3 | 4% |
Biochemistry, Genetics and Molecular Biology | 3 | 4% |
Other | 5 | 7% |
Unknown | 36 | 50% |