美国肺动脉高压患者前期治疗的现实世界依从性和持久性

IF 3 Q2 RESPIRATORY SYSTEM

Pulmonary Therapy Pub Date : 2025-09-06 DOI:10.1007/s41030-025-00311-4

Teresa De Marco, Carly J Paoli, Nicole S Croteau, Fei Tang, Harrison W Farber

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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.Results: A total of 9176 patients met the study criteria (6989 PDE5i, 1006 ERA, 1181 dual combination). After matching, each cohort included 714 patients. 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引用次数: 0

摘要

肺动脉高压（PAH）是一种罕见的进行性疾病，由肺动脉压升高导致右心室衰竭和死亡。最佳的坚持和坚持药物治疗是改善结果所必需的。本研究的目的是表征新开始治疗的患者对一线多环芳烃治疗的依从性和持久性。方法：本回顾性队列研究利用Komodo研究数据库的索赔数据。在2017年1月1日至2022年6月30日（索引日期）期间，根据≥1项磷酸二酯酶5抑制剂（PDE5i）和/或内皮素受体拮抗剂（ERA）的索赔，连续医疗和药房健康计划登记≥12个月，并包括≥1例住院或≥2例门诊肺高压/PAH索赔，以及≥1例右心导管置入术索赔，确定开始治疗的成年人。通过覆盖天数的比例（PDC）来衡量依从性；不依从性定义为PDC 60天)。各组间采用1:1:1的倾向评分匹配。结果：9176例患者符合研究标准（PDE5i 6989例，ERA 1006例，双药联合1181例）。配对后，每组纳入714例患者。ERA单药组的中位（95%置信区间）持续时间最长（26.5[19.0-33.0]个月），其次是双药联合治疗组（19.8[16.6-23.4]个月）和PDE5i单药治疗组（12.9[10.8-17.4]个月）-P = 0.019，双药联合vs ERA；P = 0.026，双重组合与PDE5i。双药联合治疗的不依从率最高（35.4%），其次是PDE5i单药治疗（17.1%）和ERA单药治疗（11.9%）——结论：初始PAH治疗的依从性不是最佳的，尤其是前期双药联合治疗。ERA单药治疗的持久性最高，其次是双药联合治疗和PDE5i单药治疗。提高依从性和持久性的策略对于优化结果至关重要。

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Real-World Adherence and Persistence of Upfront Therapy in Patients with Pulmonary Arterial Hypertension in the United States.

Introduction: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

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来源期刊

Pulmonary Therapy Medicine-Pulmonary and Respiratory Medicine

CiteScore

5.20

自引率

3.30%

发文量

审稿时长

6 weeks

期刊介绍： Aims and Scope Pulmonary Therapy is an international, open access, peer-reviewed (single-blind), and rapid publication journal. The scope of the journal is broad and will consider all scientifically sound research from pre-clinical, clinical (all phases), observational, real-world, and health outcomes research around the use of pulmonary therapies, devices, and surgical techniques. Areas of focus include, but are not limited to: asthma; chronic obstructive pulmonary disease; idiopathic pulmonary fibrosis; pulmonary hypertension; cystic fibrosis; lung cancer; respiratory tract disorders; allergic rhinitis and other respiratory allergies; influenza, pneumococcal infection, respiratory syncytial virus and other respiratory infections; and inhalers and other device therapies. The journal is of interest to a broad audience of pharmaceutical and healthcare professionals and publishes original research, reviews, case reports/series, trial protocols and short communications such as commentaries and editorials. Pulmonary Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of quality research, which may be considered of insufficient interest by other journals. Rapid Publication The journal’s publication timelines aim for a rapid peer review of 2 weeks. If an article is accepted it will be published 3–4 weeks from acceptance. The rapid timelines are achieved through the combination of a dedicated in-house editorial team, who manage article workflow, and an extensive Editorial and Advisory Board who assist with peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model this allows for the rapid, efficient communication of the latest research and reviews, fostering the advancement of pulmonary therapies. Open Access All articles published by Pulmonary Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning authors will always have an editorial contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE, GPP and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. 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