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亨廷顿舞蹈症患者对丁苯那嗪或氘代丁苯那嗪的真实世界依从性:一项回顾性数据库分析

Real-World Adherence to Tetrabenazine or Deutetrabenazine Among Patients With Huntington's Disease: A Retrospective Database Analysis.

作者信息

Claassen Daniel O, Ayyagari Rajeev, Garcia-Horton Viviana, Zhang Su, Alexander Jessica, Leo Sam

机构信息

Vanderbilt University Medical Center, 1161 21st Avenue South A-0118, Nashville, TN, 37232, USA.

Analysis Group, Inc., Boston, MA, USA.

出版信息

Neurol Ther. 2022 Mar;11(1):435-448. doi: 10.1007/s40120-021-00309-5. Epub 2021 Dec 14.

Abstract

INTRODUCTION

Chorea, a common clinical manifestation of Huntington's disease (HD), involves sudden, involuntary movements that interfere with daily functioning and contribute to the morbidity of HD. Tetrabenazine and deutetrabenazine are FDA-approved to treat chorea associated with HD. Compared to tetrabenazine, deutetrabenazine has a unique pharmacokinetic profile leading to more consistent systemic exposure, less frequent dosing, and a potentially more favorable safety/tolerability profile. Real-world adherence data for these medications are limited. Here, we evaluate real-world adherence patterns with the vesicular monoamine transporter 2 inhibitors, tetrabenazine and deutetrabenazine, among patients diagnosed with HD.

METHODS

Insurance claims data from the Symphony Health Solutions Integrated Dataverse (05/2017-05/2019) were retrospectively analyzed for patients diagnosed with HD (ICD-10-CM code G10). Patients were categorized into cohorts based on treatment. Outcomes included adherence, which was measured by proportion of days covered (PDC), adherence rate (PDC > 80%), and discontinuation rates during the 6-month follow-up period (after a 30-day dose stabilization period).

RESULTS

Patient demographic characteristics between the deutetrabenazine (N = 281) and tetrabenazine (N = 101) cohorts were comparable at baseline. Mean ± SD PDC was significantly higher in the deutetrabenazine versus tetrabenazine cohort (78.5% ± 26.7% vs. 69.3% ± 31.4%; P < 0.01). Similarly, a higher adherence rate was observed in the deutetrabenazine versus tetrabenazine cohort, though the difference was not statistically significant (64.1% vs. 55.4%; P = 0.1518). Discontinuation rates were significantly lower in the deutetrabenazine versus tetrabenazine cohort during the 6-month follow-up period (1 month, 3.5% vs. 9.2%; 3 months, 14.7% vs. 23.3%; 6 months, 25.4% vs. 37.2%; P < 0.05).

CONCLUSIONS

Results from this real-world analysis indicate that patients treated with deutetrabenazine are more adherent to treatment and have lower discontinuation rates compared with patients in the tetrabenazine cohort. However, a potential limitation is overestimated adherence, as claims for prescription fills may not capture actual use. Additional research is warranted to explore the differences in adherence patterns between treatments, which may inform treatment decision-making.

摘要

引言

舞蹈症是亨廷顿舞蹈病(HD)的常见临床表现,表现为突然的、不自主的运动,干扰日常功能并导致HD的发病。丁苯那嗪和氘代丁苯那嗪已获美国食品药品监督管理局(FDA)批准用于治疗与HD相关的舞蹈症。与丁苯那嗪相比,氘代丁苯那嗪具有独特的药代动力学特征,可导致更一致的全身暴露、给药频率更低,以及潜在更有利的安全性/耐受性特征。这些药物的真实世界依从性数据有限。在此,我们评估了被诊断为HD的患者对囊泡单胺转运体2抑制剂丁苯那嗪和氘代丁苯那嗪的真实世界依从模式。

方法

回顾性分析了Symphony Health Solutions综合数据库(2017年5月至2019年5月)中被诊断为HD(国际疾病分类第十版临床修订本代码G10)患者的保险理赔数据。根据治疗情况将患者分为不同队列。结局包括依从性,通过覆盖天数比例(PDC)、依从率(PDC>80%)以及6个月随访期(30天剂量稳定期后)的停药率来衡量。

结果

氘代丁苯那嗪队列(N = 281)和丁苯那嗪队列(N = 101)患者的人口统计学特征在基线时具有可比性。氘代丁苯那嗪队列的平均±标准差PDC显著高于丁苯那嗪队列(78.5%±26.7%对69.3%±31.4%;P<0.01)。同样,氘代丁苯那嗪队列的依从率高于丁苯那嗪队列,尽管差异无统计学意义(64.1%对55.4%;P = 0.1518)。在6个月随访期内,氘代丁苯那嗪队列的停药率显著低于丁苯那嗪队列(1个月时,3.5%对9.2%;3个月时,14.7%对23.3%;6个月时,25.4%对37.2%;P<0.05)。

结论

这项真实世界分析的结果表明,与丁苯那嗪队列的患者相比,接受氘代丁苯那嗪治疗的患者对治疗的依从性更高,停药率更低。然而,一个潜在的局限性是依从性可能被高估,因为处方配药的理赔可能无法反映实际使用情况。有必要进行更多研究以探索不同治疗之间依从模式的差异,这可能为治疗决策提供参考。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2c8e/8857359/80901c7ca163/40120_2021_309_Fig1_HTML.jpg

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