Banerji Aleena, Li Yu, Busse Paula, Riedl Marc A, Holtzman Nicole S, Li Huamin Henry, Davis-Lorton Mark, Bernstein Jonathan A, Frank Michael, Castaldo Anthony J, Long Janet, Zuraw Bruce, Lumry William, Christiansen Sandra
From the Division of Rheumatology, Allergy and Immunology, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts.
Division of Allergy and Clinical Immunology, Icahn School of Medicine at Mount Sinai, New York, New York.
Allergy Asthma Proc. 2018 May 1;39(3):212-223. doi: 10.2500/aap.2018.39.4123.
We conducted our first patient survey at the 2013 hereditary angioedema (HAE) patient summit and learned that, despite several novel therapies, the burden of disease was high.
To determine, from the patient's perspective, if any improvements in the current state of HAE care occurred over a two-year period between HAE patient summits.
A patient survey was conducted at the 2015 Hereditary Angioedema Association conference by using paper surveys that aimed at understanding the current state of HAE care. Questions included patient characteristics, burden of disease, and satisfaction with care and treatment options. Comparisons between patients with HAE with C1-inhibitor (HAE-C1INH) and patients with HAE with normal C1-inhibitor (HAE-nlC1INH), as well as between patients with HAE in 2013 and 2015, were performed by using χ2 tests.
There were 232 surveys distributed, and 143 surveys were identified as complete for inclusion and analysis from patients with self-reported HAE. Most patients had type I or type II HAE (67.5% [n = 106]), with a smaller number of patients with HAE-nlC1INH (23.6% [n = 37]). In 2015, almost half of the patients with HAE-C1INH (47.1%) and 56.7% of the patients with HAE-nlC1INH experienced a delay of ≥10 years between initial symptoms and diagnosis. Among the patients with HAE-C1INH, 25% reported one or more attacks per week and another 48% reported experiencing one or more attacks per month (fewer than one attack per week). The patients with HAE-nlC1INH reported attacks more frequently than did the patients with HAE-C1INH (p = 0.002), with 59.5% who reported attacks at least once a week. Emergency care was reported one or more times per month in 5% of the patients with HAE-C1INH and in 24.3% of the patients with HAE-nlC1INH.
Similar to 2013, although significant progress has been made, there is still a high burden of disease that faces patients with HAE.
我们在2013年遗传性血管性水肿（HAE）患者峰会上开展了首次患者调查，结果发现，尽管有几种新型疗法，但疾病负担仍然很高。
从患者的角度确定在两次HAE患者峰会之间的两年时间里，HAE护理现状是否有任何改善。
在2015年遗传性血管性水肿协会会议上进行了一项患者调查，采用纸质调查问卷，旨在了解HAE护理的现状。问题包括患者特征、疾病负担以及对护理和治疗选择的满意度。使用χ2检验对C1抑制剂缺乏型遗传性血管性水肿（HAE-C1INH）患者和C1抑制剂正常型遗传性血管性水肿（HAE-nlC1INH）患者之间，以及2013年和2015年的HAE患者之间进行比较。
共发放了232份调查问卷，其中143份被确定为完整问卷，纳入来自自我报告患有HAE的患者进行分析。大多数患者患有I型或II型HAE（67.5% [n = 106]），HAE-nlC1INH患者数量较少（23.6% [n = 37]）。2015年，几乎一半的HAE-C1INH患者（47.1%）和56.7%的HAE-nlC1INH患者在出现初始症状和确诊之间经历了≥10年的延迟。在HAE-C1INH患者中，25%的患者报告每周有一次或多次发作，另外48%的患者报告每月有一次或多次发作（每周少于一次发作）。HAE-nlC1INH患者报告发作的频率高于HAE-C1INH患者（p = 0.002），59.5%的患者报告每周至少发作一次。5%的HAE-C1INH患者和24.3%的HAE-nlC1INH患者报告每月接受一次或多次急诊治疗。
与2013年情况类似，尽管已取得显著进展，但HAE患者仍面临着较高的疾病负担。
