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从塞来昔帕格转换为口服曲前列尼尔治疗肺动脉高压的安全性和疗效:ADAPT 注册研究结果。

Safety and efficacy of transitioning from selexipag to oral treprostinil in pulmonary arterial hypertension: Findings from the ADAPT registry.

机构信息

University of Rochester Medical Center, 601 Elmwood Ave, Rochester, NY, 14642, USA.

New York-Presbyterian Brooklyn Methodist Hospital, 506 6th St, Brooklyn, NY, 11215, USA.

出版信息

Pulm Pharmacol Ther. 2023 Oct;82:102232. doi: 10.1016/j.pupt.2023.102232. Epub 2023 Jul 13.

Abstract

PURPOSE

Oral treprostinil and selexipag are drugs targeting the prostacyclin pathway and are approved for treatment of pulmonary arterial hypertension (PAH). In the setting of unsatisfactory clinical response or tolerability issues while on selexipag, there is little data on clinical benefit, safety, or strategies on transitioning to oral treprostinil. Using prospective data from the ADAPT registry, we aimed to evaluate clinical outcomes, safety, and transition strategies in ten patients with PAH transitioning from selexipag to oral treprostinil.

METHODS

ADAPT was a prospective, real-world, multicenter, United States-based registry of patients with PAH newly started on oral treprostinil, with a cohort of patients (n = 10) transitioning from selexipag to oral treprostinil. PAH variables of interest were collected from standard-of-care clinic visits. Clinical improvement was defined by modified REPLACE criterion, and risk was assessed by REVEAL Lite 2 from baseline to last follow-up. Real world transition strategies were recorded. Healthcare utilization or worsening PAH was evaluated within 30 days of transitions.

RESULTS

Seven patients transitioned due to worsening PAH or lack of efficacy on selexipag, and three patients transitioned due to tolerability issues. Based on the modified REPLACE criterion, five patients demonstrated clinical improvement after transition from selexipag to oral treprostinil. Using REVEAL Lite 2 to assess risk, three patients improved and five patients maintained risk category from baseline to last follow-up. All transitions occurred in an outpatient setting either as abrupt stop/start or cross-titration, without parenteral treprostinil bridging.

CONCLUSION

Transition from selexipag to oral treprostinil was safe, performed without parenteral prostacyclin bridging, and resulted in clinical and categorical risk improvements in some patients.

摘要

目的

口服曲前列尼尔和塞来昔帕是针对前列环素途径的药物,已被批准用于治疗肺动脉高压(PAH)。在塞来昔帕治疗时临床反应不佳或出现耐受性问题的情况下,关于转换为口服曲前列尼尔的临床获益、安全性或策略的数据很少。利用 ADAPT 登记处的前瞻性数据,我们旨在评估 10 例从塞来昔帕转换为口服曲前列尼尔的 PAH 患者的临床结局、安全性和转换策略。

方法

ADAPT 是一项前瞻性、真实世界、多中心、美国基于登记处的研究,纳入了新开始口服曲前列尼尔的 PAH 患者,其中有一组(n=10)患者从塞来昔帕转换为口服曲前列尼尔。从标准护理就诊中收集了感兴趣的 PAH 变量。根据改良的 REPLACE 标准评估临床改善,根据基线至最后随访的 REVEAL Lite 2 评估风险。记录了实际的转换策略。在转换后 30 天内评估了医疗保健利用情况或 PAH 恶化情况。

结果

7 例患者因塞来昔帕治疗的 PAH 恶化或疗效不佳而转换,3 例患者因耐受性问题而转换。根据改良的 REPLACE 标准,5 例患者在从塞来昔帕转换为口服曲前列尼尔后表现出临床改善。使用 REVEAL Lite 2 评估风险,从基线到最后随访,3 例患者风险改善,5 例患者风险类别保持不变。所有转换均在门诊环境中进行,无论是突然停药/开始还是交叉滴定,均无需静脉内曲前列尼尔桥接。

结论

从塞来昔帕转换为口服曲前列尼尔是安全的,无需静脉内前列环素桥接,并且在某些患者中可实现临床和分类风险改善。

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