Roy Anuja, Peterson Andrew, Marchant Nick, Alvir Jose, Bhambri Rahul, Lynn Jason, Benjumea Darrin, Prasad Sapna, O'Brien Alex, Chen Yong, Kemner Jason, Parasuraman Bhash
Global HEOR, Patient & Health Impact, Rare Diseases BU, Pfizer Inc, New York, NY, USA.
Department of Pharmacy Practice/Pharmacy Administration, University of the Sciences, Philadelphia, PA, USA.
Patient Prefer Adherence. 2022 Apr 29;16:1115-1129. doi: 10.2147/PPA.S352332. eCollection 2022.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a serious, underrecognized condition, which leads to heart failure and early mortality if left untreated. Until recently, heart transplantation was the only treatment for ATTR-CM. Regulatory approval of tafamidis transformed treatment for patients. In the phase 3 Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT), which established the safety and efficacy of tafamidis, medication adherence was high with 97.2% of patients taking ≥80% of scheduled doses. Evidence of real-world adherence to cardiology drugs demonstrates low adherence and suboptimal outcomes; however, real-world adherence to tafamidis has not been investigated. The main objective of this study was to describe adherence patterns of patients filling tafamidis in the Symphony Health database.
This retrospective analysis of the Symphony Health Solutions claims database used secondary adherence measures, including modified medication possession ratio (MPRm), days between fills adherence rate, and compliance rate, to assess adherence patterns of 2020 patients filling tafamidis free acid 61-mg capsules or tafamidis meglumine 4x20-mg capsules from June 1, 2019 to August 31, 2020.
Patients receiving a tafamidis formulation had characteristics consistent with the expected patient population; 71.6% were aged 75-84 years, 83.2% were male, and the highest proportion resided in the Northeast region (30.5%) of the United States. Adherence for tafamidis was high, as 75% to 100% of the patients across subgroups met or exceeded the commonly defined adherence threshold of 80%. Median number of refills ordered and received was six refills per patient. Most patients received refills with no gap (n=1633) or a gap <30 days (n=1267/1317 patients). Adherence was high across follow-up time, sex, and age subgroups. Adherence varied by geographic region, with the Northeast being significantly higher than the Midwest (mean MPRm 94.41% vs 88.21%, p=0.0007).
These results provide evidence that real-world adherence to tafamidis in patients with ATTR-CM is high.
转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）是一种严重但未得到充分认识的疾病，如果不治疗会导致心力衰竭和早期死亡。直到最近，心脏移植还是ATTR-CM的唯一治疗方法。他伏米地的监管批准改变了患者的治疗方式。在确定他伏米地安全性和有效性的3期转甲状腺素蛋白淀粉样变心肌病临床试验（ATTR-ACT）中，药物依从性很高，97.2%的患者服用了≥80%的预定剂量。心脏病药物实际依从性的证据表明依从性较低且结果不理想；然而，他伏米地的实际依从性尚未得到研究。本研究的主要目的是描述在Symphony Health数据库中服用他伏米地的患者的依从模式。
对Symphony Health Solutions索赔数据库进行的这项回顾性分析使用了二级依从性指标，包括改良药物持有率（MPRm）、两次取药间隔天数依从率和依从率，以评估2020名在2019年6月1日至2020年8月31日期间服用61毫克他伏米地游离酸胶囊或4×20毫克他伏米地葡甲胺胶囊的患者的依从模式。
接受他伏米地制剂治疗的患者特征与预期患者群体一致；71.6%的患者年龄在75 - 84岁之间，83.2%为男性，比例最高的居住在美国东北部地区（30.5%）。他伏米地的依从性很高，因为各亚组中75%至100%的患者达到或超过了通常定义的80%的依从阈值。每位患者的中位数续方次数和取药次数为6次。大多数患者取药时没有间隔（n = 1633）或间隔<30天（1317名患者中有1267名）。在随访时间、性别和年龄亚组中依从性都很高。依从性因地理区域而异，东北部地区显著高于中西部地区（平均MPRm分别为94.41%和88.21%，p = 0.0007）。
这些结果提供了证据，表明ATTR-CM患者对他伏米地的实际依从性很高。
