{"title":"遗传性血管性水肿的流行病学、经济和人文负担:系统综述。","authors":"Xin Guan, Yanan Sheng, Shuang Liu, Miao He, Tianxiang Chen, Yuxiang Zhi","doi":"10.1186/s13023-024-03265-z","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>This systematic study aims to assess the global epidemiologic, economic, and humanistic burden of illness associated with all types of hereditary angioedema.</p><p><strong>Methods: </strong>A systematic search for articles reporting the epidemiologic, economic, and humanistic burden associated with patients with HAE was conducted using English and Chinese literature databases from the inception to May 23, 2022. The selected studies were assessed for their quality and risk of bias. The study was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses and registered with the International Prospective Register of Systematic Reviews (PROSPERO; CRD42022352377).</p><p><strong>Results: </strong>In total, 65 articles that met the search inclusion criteria reported 10,310 patients with HAE, of whom 5861 were female patients. Altogether, 4312 patients (81%) and 479 patients (9%) had type 1 and type 2 HAE, respectively, whereas 422 patients (8%) had HAE-normal C1-INH. The overall prevalence of all types of HAE was between 0.13 and 1.6 cases per 100,000. The mean or median delay from the first onset of a symptom of HAE to confirmed diagnosis ranged from 3.9 to 26 years. The estimated risk of death from asphyxiation was 8.6% for patients with HAE. Hospitalization, medication, unnecessary surgeries, doctor visits, specialist services, and nursing costs are direct expenses that contribute to the growing economic burden. The indirect cost accounted mostly due to missing work ($3402/year) and loss of productivity ($5750/year). Furthermore, impairment of QoL as reported by patient-reported outcomes was observed. QoL measures identified depression, anxiety, and stress to be the most common symptoms for adult patients and children.</p><p><strong>Conclusion: </strong>This study highlights the importance of early diagnosis and the need for improving awareness among health care professionals to reduce the burden of HAE on patients and society.</p>","PeriodicalId":19651,"journal":{"name":"Orphanet Journal of Rare Diseases","volume":null,"pages":null},"PeriodicalIF":3.4000,"publicationDate":"2024-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11229247/pdf/","citationCount":"0","resultStr":"{\"title\":\"Epidemiology, economic, and humanistic burden of hereditary angioedema: a systematic review.\",\"authors\":\"Xin Guan, Yanan Sheng, Shuang Liu, Miao He, Tianxiang Chen, Yuxiang Zhi\",\"doi\":\"10.1186/s13023-024-03265-z\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>This systematic study aims to assess the global epidemiologic, economic, and humanistic burden of illness associated with all types of hereditary angioedema.</p><p><strong>Methods: </strong>A systematic search for articles reporting the epidemiologic, economic, and humanistic burden associated with patients with HAE was conducted using English and Chinese literature databases from the inception to May 23, 2022. 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引用次数: 0
摘要
背景:本系统性研究旨在评估与各种遗传性血管性水肿相关的全球流行病学、经济和人文负担:本系统性研究旨在评估与所有类型遗传性血管性水肿相关的全球流行病学、经济和人文疾病负担:方法:使用中英文文献数据库,系统检索了自开始至 2022 年 5 月 23 日期间报道与 HAE 患者相关的流行病学、经济和人文负担的文章。对所选研究的质量和偏倚风险进行了评估。研究按照《系统综述和元分析首选报告项目》进行,并在国际系统综述前瞻性注册中心(PROSPERO;CRD42022352377)进行了注册:共有 65 篇符合检索纳入标准的文章报道了 10,310 名 HAE 患者,其中 5861 人为女性患者。共有 4312 名患者(81%)和 479 名患者(9%)分别患有 1 型和 2 型 HAE,422 名患者(8%)患有 HAE 正常 C1-INH。所有类型 HAE 的总体发病率介于每 10 万人 0.13 例和 1.6 例之间。从首次出现HAE症状到确诊的平均或中位延迟时间为3.9至26年。据估计,HAE 患者死于窒息的风险为 8.6%。住院、药物治疗、不必要的手术、就诊、专科服务和护理费用等直接费用导致经济负担日益加重。间接成本主要来自缺勤(3402 美元/年)和生产力损失(5750 美元/年)。此外,根据患者报告的结果,患者的 QoL 也受到了影响。QoL测量结果显示,抑郁、焦虑和压力是成年患者和儿童最常见的症状:本研究强调了早期诊断的重要性,以及提高医护人员对减轻 HAE 对患者和社会造成的负担的认识的必要性。
Epidemiology, economic, and humanistic burden of hereditary angioedema: a systematic review.
Background: This systematic study aims to assess the global epidemiologic, economic, and humanistic burden of illness associated with all types of hereditary angioedema.
Methods: A systematic search for articles reporting the epidemiologic, economic, and humanistic burden associated with patients with HAE was conducted using English and Chinese literature databases from the inception to May 23, 2022. The selected studies were assessed for their quality and risk of bias. The study was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses and registered with the International Prospective Register of Systematic Reviews (PROSPERO; CRD42022352377).
Results: In total, 65 articles that met the search inclusion criteria reported 10,310 patients with HAE, of whom 5861 were female patients. Altogether, 4312 patients (81%) and 479 patients (9%) had type 1 and type 2 HAE, respectively, whereas 422 patients (8%) had HAE-normal C1-INH. The overall prevalence of all types of HAE was between 0.13 and 1.6 cases per 100,000. The mean or median delay from the first onset of a symptom of HAE to confirmed diagnosis ranged from 3.9 to 26 years. The estimated risk of death from asphyxiation was 8.6% for patients with HAE. Hospitalization, medication, unnecessary surgeries, doctor visits, specialist services, and nursing costs are direct expenses that contribute to the growing economic burden. The indirect cost accounted mostly due to missing work ($3402/year) and loss of productivity ($5750/year). Furthermore, impairment of QoL as reported by patient-reported outcomes was observed. QoL measures identified depression, anxiety, and stress to be the most common symptoms for adult patients and children.
Conclusion: This study highlights the importance of early diagnosis and the need for improving awareness among health care professionals to reduce the burden of HAE on patients and society.
期刊介绍:
Orphanet Journal of Rare Diseases is an open access, peer-reviewed journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on clinical trial outcome reports, either positive or negative, and articles on public health issues in the field of rare diseases and orphan drugs. The journal does not accept case reports.