De Marco Teresa, Paoli Carly J, Croteau Nicole S, Tang Fei, Farber Harrison W
Division of Cardiology, University of California San Francisco, San Francisco, CA, USA.
Johnson & Johnson Innovative Medicine, 1125 Trenton-Harbourton Road, Titusville, NJ, 08560, USA.
Pulm Ther. 2025 Sep 6. doi: 10.1007/s41030-025-00311-4.
Pulmonary arterial hypertension (PAH) is a rare, progressive disease resulting from elevated pulmonary arterial pressure leading to right ventricular failure and death. Optimal adherence and persistence to medical therapy are necessary to improve outcomes. The objective of this study was to characterize adherence and persistence to first-line PAH therapies in patients newly initiating treatment.
This retrospective cohort study utilized Komodo Research Database claims data. Adults initiating therapy were identified based on ≥ 1 claim for a phosphodiesterase 5 inhibitor (PDE5i) and/or an endothelin receptor antagonist (ERA) from January 1, 2017, to June 30, 2022 (index date), continuous medical and pharmacy health plan enrollment for ≥ 12 months before and including index, ≥ 1 inpatient or ≥ 2 outpatient claims for pulmonary hypertension/PAH, and ≥ 1 claim for right heart catheterization. Adherence was measured by proportion of days covered (PDC); nonadherence was defined as PDC < 80%. Persistence was defined as time from index to treatment discontinuation (gap in therapy > 60 days). Propensity score matching was utilized 1:1:1 across groups.
A total of 9176 patients met the study criteria (6989 PDE5i, 1006 ERA, 1181 dual combination). After matching, each cohort included 714 patients. Median (95% confidence interval) persistence was highest for ERA monotherapy (26.5 [19.0-33.0] months), followed by dual combination therapy (19.8 [16.6-23.4] months) and PDE5i monotherapy (12.9 [10.8-17.4] months)-P = 0.019, dual combination versus ERA; P = 0.026, dual combination versus PDE5i. Nonadherence was highest with dual combination therapy (35.4%), followed by PDE5i monotherapy (17.1%) and ERA monotherapy (11.9%)-P < 0.001, dual combination versus each monotherapy.
Adherence to initial PAH therapy is suboptimal, especially with upfront dual combination therapy. Persistence was highest for ERA monotherapy, followed by dual combination therapy and PDE5i monotherapy. Strategies to improve adherence and persistence are crucial to optimizing outcomes.
肺动脉高压（PAH）是一种罕见的进行性疾病，由肺动脉压力升高导致右心室衰竭和死亡。最佳的药物治疗依从性和持续性对于改善预后至关重要。本研究的目的是描述新开始治疗的患者对一线PAH治疗的依从性和持续性。
这项回顾性队列研究利用了科莫多研究数据库的索赔数据。从2017年1月1日至2022年6月30日（索引日期），根据≥1次磷酸二酯酶5抑制剂（PDE5i）和/或内皮素受体拮抗剂（ERA）的索赔记录确定开始治疗的成年人，在索引日期之前及包括索引日期在内连续参加医疗和药房健康计划≥12个月，有≥1次住院或≥2次门诊的肺动脉高压/PAH索赔记录，以及≥1次右心导管检查索赔记录。依从性通过覆盖天数比例（PDC）来衡量；不依从定义为PDC＜80%。持续性定义为从索引日期到治疗中断的时间（治疗间隔＞60天）。在各治疗组之间采用1:1:1的倾向评分匹配。
共有9176名患者符合研究标准（6989例使用PDE5i，1006例使用ERA，1181例使用双联联合治疗）。匹配后，每个队列包括714名患者。ERA单药治疗的中位（95%置信区间）持续性最高（26.5[19.0 - 33.0]个月），其次是双联联合治疗（19.8[16.6 - 23.4]个月）和PDE5i单药治疗（12.9[10.8 - 17.4]个月）——双联联合治疗与ERA相比，P = 0.019；双联联合治疗与PDE5i相比，P = 0.026。双联联合治疗的不依从率最高（3
