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Abstract：
BACKGROUND: Real-world data on the comparative effectiveness of endothelin receptor antagonists (ERAs; macitentan, bosentan, ambrisentan) for pulmonary arterial hypertension (PAH), particularly in Asian countries, are scarce. We evaluated the persistence of these ERAs before and after macitentan approval in Japan (2015).
METHODS: We used real-world data from the Japanese Medical Data Vision administrative claims database between April 2008 and November 2020. Patients with PAH were identified from the dataset. Persistence to ERA treatment before and after approval of macitentan in Japan was defined as the time between start of the index ERA and treatment discontinuation or death. Propensity score adjustment was applied to minimize confounding effects among treatment groups.
RESULTS: In the pre-macitentan approval cohort, 153 and 51 patients received bosentan and ambrisentan, respectively. In the post-macitentan approval cohort, 331, 284, and 91 patients received macitentan, bosentan, and ambrisentan, respectively. Unadjusted median persistence for ambrisentan- and bosentan-treated patients was 19 and 10 months, respectively (adjusted HR 0.87 [95% CI 0.61-1.24]; P = 0.434 [bosentan as reference]). In the post-macitentan approval cohort, unadjusted median persistence was 18 months for macitentan-treated patients versus 6 and 8 months for ambrisentan- and bosentan-treated patients, respectively. Adjusted HRs for ambrisentan and bosentan were 1.48 (95% CI 1.12-1.95; P = 0.006) and 1.63 (95% CI 1.30-2.04; P < 0.001 [macitentan as reference]), respectively.
CONCLUSIONS: Real-world data for Japanese patients with PAH showed that persistence was significantly higher for macitentan, versus ambrisentan and bosentan, since its approval.
METHODS: We used real-world data from the Japanese Medical Data Vision administrative claims database between April 2008 and November 2020. Patients with PAH were identified from the dataset. Persistence to ERA treatment before and after approval of macitentan in Japan was defined as the time between start of the index ERA and treatment discontinuation or death. Propensity score adjustment was applied to minimize confounding effects among treatment groups.
RESULTS: In the pre-macitentan approval cohort, 153 and 51 patients received bosentan and ambrisentan, respectively. In the post-macitentan approval cohort, 331, 284, and 91 patients received macitentan, bosentan, and ambrisentan, respectively. Unadjusted median persistence for ambrisentan- and bosentan-treated patients was 19 and 10 months, respectively (adjusted HR 0.87 [95% CI 0.61-1.24]; P = 0.434 [bosentan as reference]). In the post-macitentan approval cohort, unadjusted median persistence was 18 months for macitentan-treated patients versus 6 and 8 months for ambrisentan- and bosentan-treated patients, respectively. Adjusted HRs for ambrisentan and bosentan were 1.48 (95% CI 1.12-1.95; P = 0.006) and 1.63 (95% CI 1.30-2.04; P < 0.001 [macitentan as reference]), respectively.
CONCLUSIONS: Real-world data for Japanese patients with PAH showed that persistence was significantly higher for macitentan, versus ambrisentan and bosentan, since its approval.
摘要：
背景：关于内皮素受体拮抗剂（ERA；Macitentan，波生坦,ambrisentan）用于肺动脉高压（PAH），特别是在亚洲国家，是稀缺的。我们评估了日本Macitentan批准前后这些ERA的持久性（2015年）。
方法：我们在2008年4月至2020年11月期间使用了来自日本医疗数据视觉行政索赔数据库的真实数据。从数据集中鉴定患有PAH的患者。在日本批准Macitentan之前和之后，ERA治疗的持久性定义为ERA指数开始与治疗中止或死亡之间的时间。应用倾向评分调整以最小化治疗组之间的混杂效应。
结果：在Macitentan批准之前的队列中，153名和51名患者接受了波生坦和ambrisentan，分别。在Macitentan批准后的队列中，331、284和91名患者接受马西坦治疗，波生坦,还有ambrisentan,分别。安利芬太尼和波生坦治疗患者的未调整的中位持续时间为19个月和10个月，分别（调整后的HR0.87[95%CI0.61-1.24]；P=0.434[波生坦作为参考]）。在Macitentan批准后的队列中，未调整的中位持续时间为18个月的马西坦治疗的患者与6个月和8个月的安利芬太尼和波生坦治疗的患者，分别。Ambrisentan和Bosentan的调整后的HR分别为1.48（95％CI1.12-1.95；P=0.006）和1.63（95％CI1.30-2.04；P<0.001[Macitentan作为参考]），分别。
结论：日本PAH患者的真实世界数据显示，马西坦的持久性明显更高，对抗ambrisentan和Bosentan,自批准以来。
方法：我们在2008年4月至2020年11月期间使用了来自日本医疗数据视觉行政索赔数据库的真实数据。从数据集中鉴定患有PAH的患者。在日本批准Macitentan之前和之后，ERA治疗的持久性定义为ERA指数开始与治疗中止或死亡之间的时间。应用倾向评分调整以最小化治疗组之间的混杂效应。
结果：在Macitentan批准之前的队列中，153名和51名患者接受了波生坦和ambrisentan，分别。在Macitentan批准后的队列中，331、284和91名患者接受马西坦治疗，波生坦,还有ambrisentan,分别。安利芬太尼和波生坦治疗患者的未调整的中位持续时间为19个月和10个月，分别（调整后的HR0.87[95%CI0.61-1.24]；P=0.434[波生坦作为参考]）。在Macitentan批准后的队列中，未调整的中位持续时间为18个月的马西坦治疗的患者与6个月和8个月的安利芬太尼和波生坦治疗的患者，分别。Ambrisentan和Bosentan的调整后的HR分别为1.48（95％CI1.12-1.95；P=0.006）和1.63（95％CI1.30-2.04；P<0.001[Macitentan作为参考]），分别。
结论：日本PAH患者的真实世界数据显示，马西坦的持久性明显更高，对抗ambrisentan和Bosentan,自批准以来。
