坚持使用囊性纤维化跨膜传导调节剂 (CFTR) 调节剂:全国专科药房数据库分析。

IF 2.4
Zumi Mehta, Khalid M Kamal, Richard Miller, Jordan R Covvey, Vincent Giannetti
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引用次数: 0

摘要

背景:随着囊性纤维化跨膜传导调节器(CFTR)调节剂的问世,囊性纤维化(CF)治疗取得了重大进展。坚持用药是治疗囊性纤维化的一个重要目标,因为不坚持用药会导致不良的健康后果:利用全国专科药房数据库计算服用 CFTR 调节剂患者的用药依从性:这项回顾性观察队列研究利用 2017 年 9 月至 2018 年 8 月期间去标识化的专科药房数据,评估三种 CFTR 调节剂的用药依从性:ivacaftor、lumacaftor/ivacaftor 和 tezacaftor/ivacaftor & ivacaftor。主要结果是每种药物的覆盖天数比例(PDC),并比较不同年龄组和保险特征的平均 PDC 值。所有分析均使用 SAS 9.4 大学版(SAS Institute,Cary,NC)进行:共对2548名患者进行了分析,其中1289名(50.59%)患者使用lumacaftor/ivacaftor,784名(30.77%)患者使用ivacaftor,475名(18.64%)患者使用tezacaftor/ivacaftor & ivacaftor。所有 CFTR 调节剂的平均 PDC 值均高于 0.80。特扎卡夫托/伊伐卡夫托和伊伐卡夫托的总体PDC值最高,为0.92,而鲁马卡夫托/伊伐卡夫托和伊伐卡夫托的PDC值均为0.84。与成人相比,使用 lumacaftor/ivacaftor (p = 0.0001) 和 tezacaftor/ivacaftor & ivacaftor (p = 0.001) 的儿童/青少年的平均 PDC 值显著较高,但 ivacaftor (p = 0.3744)则不然。不同保险特征的 PDC 无统计学差异:据我们所知,这是第一项利用大型全国性专科数据库评估三种 CFTR 调节剂依从性的研究。由于 CFTR 调节剂疗法的购买成本较高,因此有必要提高 CF 患者的依从率。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Adherence to cystic fibrosis transmembrane conductance regulator (CFTR) modulators: analysis of a national specialty pharmacy database.

Adherence to cystic fibrosis transmembrane conductance regulator (CFTR) modulators: analysis of a national specialty pharmacy database.

Adherence to cystic fibrosis transmembrane conductance regulator (CFTR) modulators: analysis of a national specialty pharmacy database.

Background: There have been significant advances in Cystic Fibrosis (CF) treatment, with the introduction of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators. Adherence is an important goal for CF management, as nonadherence is linked to poor health outcomes.

Objective: To calculate the medication adherence in patients taking CFTR modulators using a national specialty pharmacy database.

Methods: This retrospective observational cohort study utilized de-identified specialty pharmacy data from September 2017 to August 2018 to assess medication adherence for three CFTR modulators: ivacaftor, lumacaftor/ivacaftor, and tezacaftor/ivacaftor & ivacaftor. The primary outcome was proportion of days covered (PDC) for each medication, with mean PDC values compared across age groups and insurance characteristics. All analyses were performed using the SAS 9.4 University Edition (SAS Institute, Cary, NC).

Results: A total of 2,548 patients were analyzed, including 1,289 (50.59%) patients on lumacaftor/ivacaftor, 784 (30.77%) on ivacaftor, and 475 (18.64%) on tezacaftor/ivacaftor & ivacaftor. The mean PDC value for all CFTR modulators was above 0.80. Tezacaftor/ivacaftor & ivacaftor had the highest overall PDC of 0.92, while PDC values for both lumacaftor/ivacaftor and ivacaftor were 0.84. Children/adolescents on lumacaftor/ivacaftor (p = 0.0001) and tezacaftor/ivacaftor & ivacaftor (p = 0.001) had significantly higher mean PDC values compared to adults but not for ivacaftor (p = 0.3744). No statistical differences were seen in PDC across insurance characteristics.

Conclusion: To the best of our knowledge, this is the first study to assess the adherence of three CFTR modulators using a large nationwide specialty database. With high acquisition costs of CFTR modulator therapies, there is a need to improve rates of adherence in patients with CF.

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来源期刊
Journal of Drug Assessment
Journal of Drug Assessment PHARMACOLOGY & PHARMACY-
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