Safety and effectiveness of riociguat for chronic thromboembolicpulmonary hypertension in real-world clinical practice: interim data frompost-marketing surveillance in Japan

摘要

This multicenter, prospective, non-interventional study (ClinicalTrials.gov:{"type":"clinical-trial","attrs":{"text":"NCT02117791","term_id":"NCT02117791"}}NCT02117791) evaluated the safety and effectiveness of riociguat for chronicthromboembolic pulmonary hypertension in Japanese clinical practice, registeringall patients with chronic thromboembolic pulmonary hypertension treated withriociguat following its launch in Japan in April 2014. Safety was assessed byanalyzing the adverse drug reactions. Effectiveness measurements included theassessment of change in World Health Organization functional class, six-minutewalk test, and hemodynamics. Overall, 1031 patients were included in the safetyanalysis with 811 (78.7%) patients in World Health Organization functional classII/III. The mean treatment duration was 591.4 days (median 441.0 days). Adversedrug reactions were reported in 19.5% of patients, the most common beinghypotension (5.9%), headache (3.0%), dizziness (1.9%), and gastroesophagealreflux disease (1.5%). Serious adverse drug reactions were reported in 2.1% ofpatients. Estimated survival was 97.0% at one year, 95.8% at two years, and94.4% at three years. The effectiveness analysis (n = 1027)showed significant increases from baseline in six-minute walking distance, andsignificant reductions from baseline in mean pulmonary arterial pressure andpulmonary vascular resistance. These interim results of riociguat in Japanesepatients with chronic thromboembolic pulmonary hypertension demonstrated asafety profile that was generally consistent with those of pivotal clinicalstudies. The study is ongoing, and will continue to provide insights into thesafety and effectiveness of riociguat in real-world practice.