School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, 211198, China.
Medical Affairs, Takeda (China) International Trading Company, Beijing, 100006, China.
Orphanet J Rare Dis. 2024 Jul 8;19(1):256. doi: 10.1186/s13023-024-03265-z.
This systematic study aims to assess the global epidemiologic, economic, and humanistic burden of illness associated with all types of hereditary angioedema.
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).
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.
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.
本系统研究旨在评估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。
使用英文和中文文献数据库，从建立到 2022 年 5 月 23 日，对报道与 HAE 患者相关的流行病学、经济和人文疾病负担的文章进行了系统搜索。对所选研究进行了质量和偏倚风险评估。本研究按照系统评价和荟萃分析的首选报告项目进行，并在国际前瞻性系统评价注册库（PROSPERO；CRD42022352377）中进行了注册。
共有 65 篇符合搜索纳入标准的文章报道了 10310 例 HAE 患者，其中 5861 例为女性患者。共有 4312 例（81%）和 479 例（9%）患者分别患有 1 型和 2 型 HAE，而 422 例（8%）患者患有 HAE-正常 C1-INH。所有类型 HAE 的总体患病率在每 10 万人中有 0.13 至 1.6 例。从 HAE 症状首次发作到确诊的平均或中位数延迟时间为 3.9 至 26 年。HAE 患者窒息死亡的风险估计为 8.6%。住院、药物、不必要的手术、就诊、专科服务和护理费用是导致经济负担不断增加的直接费用。间接费用主要归因于缺勤（每年 3402 美元）和生产力损失（每年 5750 美元）。此外，还观察到患者报告结局报告的 QoL 受损。QoL 测量确定抑郁、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，并需要提高卫生保健专业人员的认识，以减轻 HAE 给患者和社会带来的负担。
