Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, São Paulo, Brazil.
Division of Medical Genetics, Department of Biomedicine, University of Basel, Basel, Switzerland.
Int Arch Allergy Immunol. 2021;182(8):697-708. doi: 10.1159/000513896. Epub 2021 Mar 3.
Prevention of attacks is a major goal in management of patients with hereditary angioedema (HAE). We aimed to investigate the effects of a systematic intervention for HAE patients.
Thirty-three patients with HAE with C1-inhibitor deficiency, belonging to a single family, participated in a management program coordinated by an allergist/immunologist. Angioedema attacks before intervention were ascertained by interviews and emergency room charts and recorded prospectively by patients or caregivers after enrollment. Mean number of attacks/month was compared at 12 months preintervention and 8 and 14 months within intervention. Patient-reported outcome instruments were used to assess quality of life, including HAE Quality of Life (HAE-QoL) questionnaire, psychological conditions, and work impairment, at baseline and 8 and 14 months within intervention. Data were stored in REDCap platform and analyzed by adjusted Bayesian models of double Poisson regression.
Mean number of attacks/month significantly decreased (95% credible interval [CrI] excluding 0) from 1.15 preintervention to 0.25 and 0.23, 8 and 14 months within intervention, with mean decreases of -0.89 (95% CrI: -1.21 to -0.58) and -0.92 (95% CrI: -1.22 to -0.60), respectively. HAE-QoL scores showed mean total increases of 15.2 (95% CrI: 1.23-29.77) and 26 (95% CrI: 14.56-39.02) at 8 and 14 months within the study, as compared to baseline, revealing marked improvement in quality of life. Significant increase in role-emotional and reduction of depression, stress, and anxiety were observed at 14 months.
A systematic approach integrating HAE-specific care with effective handling of psychological issues decreased the number of attacks and improved quality of life, targets for best practice in HAE.
预防发作是遗传性血管性水肿（HAE）患者管理的主要目标。我们旨在研究系统性干预对 HAE 患者的影响。
33 名 C1 抑制剂缺乏症的 HAE 患者参与了由过敏症专家/免疫学家协调的管理计划。通过访谈和急诊室图表确定干预前的血管性水肿发作，并在登记后由患者或护理人员进行前瞻性记录。比较干预前 12 个月、干预内 8 个月和 14 个月的平均每月发作次数。使用 HAE 生活质量（HAE-QoL）问卷、心理状况和工作障碍等患者报告的结果工具，在基线以及干预内 8 个月和 14 个月评估生活质量。数据存储在 REDCap 平台中，并通过双泊松回归的调整贝叶斯模型进行分析。
每月发作次数的平均值（95%可信区间[CrI]不包括 0）从干预前的 1.15 显著减少到 0.25 和 0.23，分别为干预内 8 个月和 14 个月，平均减少量为 -0.89（95%CrI：-1.21 至-0.58）和 -0.92（95%CrI：-1.22 至-0.60）。与基线相比，HAE-QoL 评分在研究的 8 个月和 14 个月内分别显示出平均总分增加 15.2（95%CrI：1.23-29.77）和 26（95%CrI：14.56-39.02），表明生活质量得到了显著改善。在 14 个月时观察到角色情感显著增加，抑郁、压力和焦虑减少。
整合 HAE 特异性护理和有效处理心理问题的系统方法减少了发作次数并改善了生活质量，这是 HAE 最佳实践的目标。
