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Riociguat treatment in patients with pulmonary arterial hypertension: Final safety data from the EXPERT registry
Respiratory Medicine ( IF 3.5 ) Pub Date : 2020-12-03 , DOI: 10.1016/j.rmed.2020.106241
Marius M Hoeper 1 , Miguel-Angel Gomez Sanchez 2 , Marc Humbert 3 , David Pittrow 4 , Gérald Simonneau 5 , Henning Gall 6 , Ekkehard Grünig 7 , Hans Klose 8 , Michael Halank 9 , David Langleben 10 , Repke J Snijder 11 , Pilar Escribano Subias 12 , Lisa M Mielniczuk 13 , Tobias J Lange 14 , Jean-Luc Vachiéry 15 , Hubert Wirtz 16 , Douglas S Helmersen 17 , Iraklis Tsangaris 18 , Joan A Barberà 19 , Joanna Pepke-Zaba 20 , Anco Boonstra 21 , Stephan Rosenkranz 22 , Silvia Ulrich 23 , Regina Steringer-Mascherbauer 24 , Marion Delcroix 25 , Pavel Jansa 26 , Iveta Šimková 27 , George Giannakoulas 28 , Jens Klotsche 29 , Evgenia Williams 30 , Christian Meier 30 , Hossein-Ardeschir Ghofrani 6 ,
Affiliation  

Objective

The soluble guanylate cyclase stimulator riociguat is approved for the treatment of adult patients with pulmonary arterial hypertension (PAH) and inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension following Phase 3 randomized trials. The EXPosurE Registry RiociguaT in patients with pulmonary hypertension (EXPERT) study was designed to monitor the long-term safety of riociguat in clinical practice.

Methods

EXPERT was an international, multicenter, prospective, uncontrolled, non-interventional cohort study of patients treated with riociguat. Patients were followed for at least 1 year and up to 4 years from enrollment or until 30 days after stopping riociguat treatment. Primary safety outcomes were adverse events (AEs) and serious adverse events (SAEs) coded using Medical Dictionary for Regulatory Activities preferred terms and System Organ Classes version 21.0, collected during routine clinic visits (usually every 3–6 months) and collated via case report forms.

Results

In total, 326 patients with PAH were included in the analysis. The most common AEs in these patients were dizziness (11.7%), right ventricular (RV)/cardiac failure (10.7%), edema/peripheral edema (10.7%), diarrhea (8.6%), dyspnea (8.0%), and cough (7.7%). The most common SAEs were RV/cardiac failure (10.1%), pneumonia (6.1%), dyspnea (4.0%), and syncope (3.4%). The exposure-adjusted rate of hemoptysis/pulmonary hemorrhage was 2.5 events per 100 patient-years.

Conclusion

Final data from EXPERT show that in patients with PAH, the safety of riociguat in clinical practice was consistent with clinical trials, with no new safety concerns identified and a lower exposure-adjusted rate of hemoptysis/pulmonary hemorrhage than in the long-term extension of the Phase 3 trial in PAH.



中文翻译:

利奥西呱治疗肺动脉高压患者:来自 EXPERT 登记处的最终安全性数据

客观的

经过 3 期随机试验,可溶性鸟苷酸环化酶刺激剂利奥西呱被批准用于治疗患有肺动脉高压 (PAH) 和无法手术或持续/复发性慢性血栓栓塞性肺动脉高压的成年患者。EXPosurE注册利奥西呱在肺动脉高压患者中的研究(EXPERT)旨在监测利奥西呱在临床实践中的长期安全性。

方法

EXPERT 是一项针对利奥西呱治疗患者的国际、多中心、前瞻性、非对照、非干预性队列研究。自入组起或停止利奥西呱治疗后 30 天,对患者进行至少 1 年、最长 4 年的随访。主要安全性结局是使用监管活动医学词典首选术语和系统器官分类 21.0 版编码的不良事件 (AE) 和严重不良事件 (SAE),在常规临床就诊期间(通常每 3-6 个月)收集并通过病例报告进行整理形式。

结果

分析中总共纳入了 326 名 PAH 患者。这些患者中最常见的 AE 是头晕(11.7%)、右心室(RV)/心力衰竭(10.7%)、水肿/外周水肿(10.7%)、腹泻(8.6%)、呼吸困难(8.0%)和咳嗽(7.7%)。最常见的 SAE 是右心室/心力衰竭 (10.1%)、肺炎 (6.1%)、呼吸困难 (4.0%) 和晕厥 (3.4%)。咯血/肺出血的暴露调整率为每 100 患者年 2.5 次。

结论

EXPERT的最终数据显示,在PAH患者中,利奥西呱在临床实践中的安全性与临床试验一致,没有发现新的安全问题,并且与长期延长治疗相比,暴露调整后的咯血/肺出血发生率较低。 PAH 的 3 期试验。

更新日期:2021-01-08
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