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在日本,甲状腺素运载蛋白淀粉样变心肌病患者服用塔法米迪的依从性和持久性。

Tafamidis medication adherence and persistence in patients with transthyretin amyloid cardiomyopathy in Japan.

机构信息

Department of Cardiovascular Medicine, Institute for Advancement of Clinical and Translational Science, Kyoto University Hospital, Kyoto, Japan.

Pfizer Portugal, Porto Salvo, Portugal.

出版信息

ESC Heart Fail. 2024 Oct;11(5):2881-2888. doi: 10.1002/ehf2.14736. Epub 2024 May 23.

Abstract

AIMS

This study aimed to describe baseline characteristics and adherence among patients with transthyretin amyloid cardiomyopathy (ATTR-CM) treated with tafamidis (VYNDAQEL®) in Japan using the Japanese Medical Data Vision (MDV) database.

METHODS AND RESULTS

This study was a non-interventional, retrospective cohort study of adult (≥18 years old) patients in the Japanese MDV claims database diagnosed with ATTR-CM and with at least two tafamidis prescriptions of dose strength 4 × 20 mg/day between 1 March 2019 and 31 August 2021. The date of the first prescription was defined as the index date, with follow-up time defined as the time between the first and last prescription plus the days' supply from the last refill. Baseline characteristics were assessed during a 12 month pre-index period. Adherence was measured using two metrics: (i) the modified medication possession ratio (mMPR), calculated by taking the sum of days supplied for all fills within the follow-up period, divided by the number of days of follow-up, and reported as a percentage, with patients classified as adherent with an mMPR of ≥80%, and (ii) the proportion of days covered (PDC), calculated by taking the total number of days' supply dispensed during the follow-up period divided by the number of days of follow-up, adjusting for any days' supply overlap. A total of 210 patients were identified; the mean (standard deviation) age of the cohort was 77 (5.9) years, and the majority (89%) were male. The most common baseline cardiovascular comorbidities were heart failure (85%), ischaemic heart disease (66%), hypertensive diseases (49%), and diabetes (35%); 75% of patients received heart failure medications in the 12 months prior to index, with the most common being beta-blockers (49%), diuretics (48%), angiotensin receptor blockers (30%), angiotensin-converting enzyme inhibitors (22%), and sodium-glucose cotransporter-2 inhibitors (8.1%). Over an average 14 month follow-up, mean mMPR was 96% with a median of 100% [inter-quartile range (IQR): 97-101%]; 93% of patients were adherent (defined as an mMPR ≥ 80%). In the same follow-up period, mean PDC was 93.6% with a median of 99% (IQR: 93-100%). Persistence was high with 78% of patients having a 0 day gap between prescription refills.

CONCLUSIONS

This study found high adherence rates to tafamidis in this real-world Japanese patient population. Adherence rates in this study were similar to those reported by the tafamidis clinical trial and a previously published US commercial claims adherence analysis. Further studies should be conducted to assess the impact of real-world adherence on real-world outcomes.

摘要

目的

本研究旨在使用日本医疗数据视觉(MDV)数据库描述接受他司美坦(VYNDAQEL®)治疗的转甲状腺素淀粉样变性心肌病(ATTR-CM)患者的基线特征和依从性。

方法和结果

这是一项在日本 MDV 索赔数据库中进行的非干预性、回顾性队列研究,纳入了年龄≥18 岁的成年(≥18 岁)患者,这些患者在 2019 年 3 月 1 日至 2021 年 8 月 31 日期间被诊断为 ATTR-CM,且至少有两次他司美坦的处方剂量为 4×20mg/天。第一次处方的日期定义为索引日期,随访时间定义为第一次和最后一次处方之间的时间加上最后一次续药的天数。在索引前 12 个月评估了基线特征。采用两种指标衡量依从性:(i)改良用药比例(mMPR),通过将随访期间所有剂量的供应天数相加,除以随访天数,以百分比表示,mMPR≥80%的患者被归类为依从性好;(ii)覆盖率(PDC),通过将随访期间的总供药天数除以随访天数来计算,同时调整供药重叠天数。共确定了 210 名患者;队列的平均(标准差)年龄为 77(5.9)岁,大多数(89%)为男性。最常见的基线心血管合并症是心力衰竭(85%)、缺血性心脏病(66%)、高血压疾病(49%)和糖尿病(35%);75%的患者在索引前 12 个月内接受了心力衰竭药物治疗,最常见的药物是β受体阻滞剂(49%)、利尿剂(48%)、血管紧张素受体阻滞剂(30%)、血管紧张素转换酶抑制剂(22%)和钠-葡萄糖共转运蛋白 2 抑制剂(8.1%)。在平均 14 个月的随访期间,mMPR 平均值为 96%,中位数为 100%[四分位距(IQR):97-101%];93%的患者依从性良好(定义为 mMPR≥80%)。在相同的随访期间,平均 PDC 为 93.6%,中位数为 99%(IQR:93-100%)。患者的持久性较高,有 78%的患者在处方续药之间没有 0 天的间隔。

结论

本研究发现,在日本真实世界的患者群体中,他司美坦的依从率较高。本研究中的依从率与他司美坦临床试验和之前发表的美国商业索赔依从性分析报告中的结果相似。应进一步开展研究,评估真实世界中的依从性对真实世界结局的影响。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4963/11424365/3d9dea5bf3c4/EHF2-11-2881-g001.jpg

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