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西班牙梗阻性肥厚型心肌病患者的临床管理与医疗资源利用:一项真实世界研究

Clinical management and healthcare resource utilization among patients with obstructive hypertrophic cardiomyopathy in Spain: a real-world study.

作者信息

Barriales-Villa Roberto, Escobar-López Luis, Vilanova Larena David, Salazar-Mendiguchía Joel, Echeto Ainara, Hernández Ignacio, Rebollo-Gómez Elena, Gimeno Juan Ramón

机构信息

Unidad de Cardiopatías Familiares, Servicio de Cardiología, Complexo Hospitalario Universitario A Coruña, Instituto de Investigación Biomédica de A Coruña (INIBIC), A Coruña, Spain; Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares (CIBERCV), Instituto de Salud Carlos III, Madrid, Spain.

Medical Department, Bristol-Myers Squibb, Madrid, Spain.

出版信息

Rev Esp Cardiol (Engl Ed). 2025 May 5. doi: 10.1016/j.rec.2025.04.004.

Abstract

INTRODUCTION AND OBJECTIVES

Obstructive hypertrophic cardiomyopathy (oHCM), whose symptoms range from dyspnea to heart failure or sudden cardiac death, accounts for approximately 70% of all hypertrophic cardiomyopathy cases. This study aimed to analyze the lack of comprehensive data on oHCM management and determine its clinical and economic burden in Spain.

METHODS

This retrospective observational study, based on electronic medical records (BIG-PAC), enrolled adults from 1 January 2014, to 31 October 2022, from the time of HCM diagnosis. The analysis focused on epidemiology, patient characteristics and management, transitions between New York Heart Association (NYHA) functional classes, healthcare resource utilization (HCRU), and associated costs.

RESULTS

A total of 752 oHCM patients were included (mean age: 63 years; male: 57.6%). NYHA functional classification at diagnosis was as follows: 12% NYHA-I, 47.9% NYHA-II, 31.5% NYHA-III, and 8.6% NYHA-IV. The prevalence of HCM and oHCM was 28 and 7 per 10 000 individuals, respectively. Patients received a mean of 2.4 (SD 1.5) treatments, mainly beta-blockers. Only patients in NYHA classes III and IV underwent septal reduction therapies (SRT) (13.1% and 47.7%, respectively); 38.7% and 35.5% of NYHA-III and -IV patients who received SRT, respectively, improved to a lower NYHA class. Symptom severity, as measured by NYHA class, was associated with increased rates of hospitalization, cardiovascular events, mortality, and higher HCRU and costs. Mean annualized, direct, adjusted health care costs ranged from €4142 (95%CI: €3110-€5175) in NYHA-I to €16 677 (95%CI: €15 482-€17 872) in NYHA-IV.

CONCLUSIONS

This is the first Spanish study to evaluate oHCM patient management and to demonstrate its impact in terms of increased hospitalizations, mortality, HCRU, and healthcare costs, trends that parallel the progression of symptoms by NYHA functional class. Patients who underwent SRT showed partial symptom improvement.

摘要

引言与目的

梗阻性肥厚型心肌病(oHCM)的症状从呼吸困难到心力衰竭或心源性猝死不等,约占所有肥厚型心肌病病例的70%。本研究旨在分析oHCM管理方面缺乏全面数据的情况,并确定其在西班牙的临床和经济负担。

方法

这项基于电子病历(BIG-PAC)的回顾性观察研究纳入了自2014年1月1日至2022年10月31日确诊为HCM的成年患者。分析重点在于流行病学、患者特征与管理、纽约心脏协会(NYHA)心功能分级之间的转换、医疗资源利用(HCRU)以及相关成本。

结果

共纳入752例oHCM患者(平均年龄:63岁;男性:57.6%)。诊断时的NYHA心功能分级如下:NYHA-I级占12%,NYHA-II级占47.9%,NYHA-III级占31.5%,NYHA-IV级占8.6%。HCM和oHCM的患病率分别为每10000人28例和7例。患者平均接受2.4种(标准差1.5)治疗,主要是β受体阻滞剂。只有NYHA III级和IV级的患者接受了室间隔减容治疗(SRT)(分别为13.1%和47.7%);接受SRT的NYHA-III级和-IV级患者中,分别有38.7%和35.5%的心功能改善至较低级别。以NYHA分级衡量的症状严重程度与住院率、心血管事件、死亡率增加以及更高的HCRU和成本相关。平均年化、直接、调整后的医疗费用从NYHA-I级的4142欧元(95%CI:3110欧元 - 5175欧元)到NYHA-IV级的16677欧元(95%CI:15482欧元 - 17872欧元)不等。

结论

这是西班牙第一项评估oHCM患者管理并证明其在住院率、死亡率、HCRU和医疗成本增加方面影响的研究,这些趋势与NYHA心功能分级的症状进展情况相似。接受SRT的患者症状有部分改善。

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