Praxis Precision Medicines, 99 High Street, 30th Floor, Boston, MA, 02110, USA.
University of Kansas Medical Center, Kansas City, KS, 66160, USA.
Adv Ther. 2022 Dec;39(12):5546-5567. doi: 10.1007/s12325-022-02318-8. Epub 2022 Oct 14.
Essential tremor (ET) affects approximately 7 million people in the USA, yet public recognition of the disease and its impact remain low.
A retrospective observational study examined US claims data from 2015 to 2019 using the Compile database. ET diagnoses were captured using longitudinal data from 2015 to 2019 and for the year 2019, with diagnosis estimates extrapolated to the general US population. Confirmed ET was identified by an ET diagnosis code with at least two relevant prescriptions or by two diagnosis codes for ET and unspecified tremor at least 90 days apart. Comorbidity and treatment use data were extracted, and medication compliance and 2-year treatment persistence were assessed as measures of treatment adherence.
A total of 1,336,183 patients with ET diagnoses codes were identified from 2015 through 2019, corresponding to 2,226,971 projected US diagnoses. In 2019, 128,263 patients had a confirmed ET diagnosis, corresponding to 213,772 projected US confirmed diagnoses. Of these, 96% had at least one comorbidity, and 64% received at least one pharmacologic treatment. Propranolol (24%) and primidone (20%) comprised the most common ET prescriptions. Two-year medication discontinuation rates were approximately 40%.
Our findings revealed that 1 million people were diagnosed and sought treatment for ET in the USA from 2015 to 2019. Projected population estimates of approximately 2 million people diagnosed suggest a further 1 million remain untreated. Our findings highlight the complexity of patient care in ET, complicated by delayed diagnoses, multiple comorbidities, and lack of effective and tolerable therapies that can mitigate treatment adherence limitations.
特发性震颤（ET）影响了美国约 700 万人，但公众对该病的认识及其影响仍然很低。
本回顾性观察性研究使用 Compile 数据库，对 2015 年至 2019 年的美国索赔数据进行了分析。ET 诊断通过 2015 年至 2019 年的纵向数据和 2019 年的数据进行捕捉，并将诊断估计值外推至美国一般人群。通过至少有两个相关处方的 ET 诊断代码或至少相隔 90 天的两个 ET 和未特指震颤的诊断代码来确定确诊的 ET。提取合并症和治疗使用数据，并评估药物依从性和 2 年治疗持久性作为治疗依从性的衡量标准。
2015 年至 2019 年期间，共从 ET 诊断代码中确定了 1336183 例患者，相当于预测的 2226971 例美国诊断。2019 年，有 128263 例患者确诊为 ET，相当于预测的美国确诊患者 213772 例。其中，96%的患者至少有一种合并症，64%的患者接受了至少一种药物治疗。普萘洛尔（24%）和扑米酮（20%）是最常见的 ET 处方。两年停药率约为 40%。
我们的研究结果表明，2015 年至 2019 年期间，美国有 100 万人被诊断为 ET 并接受了治疗。预计约有 200 万人被诊断为该病，这表明还有 100 万人未得到治疗。我们的研究结果强调了 ET 患者护理的复杂性，这是由诊断延迟、多种合并症以及缺乏有效的、可耐受的治疗方法导致治疗依从性受限所导致的。
