McLaughlin, Vallerie V.Jansa, PavelNielsen-Kudsk, Jens E.Halank, MichaelSimonneau, GéraldGrünig, EkkehardUlrich, SilviaRosenkranz, StephanGómez Sánchez, Miguel A.Pulido, TomásPepke-Zaba, JoannaBarberà i Mir, Joan AlbertHoeper, Marius MVachiéry, Jean-LucLang, IreneCarvalho, FrancineMeier, ChristianMueller, KatharinaNikkho, SylviaD'Armini, Andrea M.2018-06-252018-06-252017-12-281932-6203https://hdl.handle.net/2445/123223Background: 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.9 p.application/pdfengcc-by (c) McLaughlin, Vallerie V. et al., 2017http://creativecommons.org/licenses/by/3.0/esHipertensió pulmonarMalalties de l'aparell respiratoriAssaigs clínicsPulmonary hypertensionRespiratory organs diseasesClinical trialsinfo:eu-repo/semantics/article6785282018-06-25info:eu-repo/semantics/openAccess29282032