Squeglia Veronica, Barbarino Alessandro, Bova Maria, Gravante Carmela, Petraroli Angelica, Spadaro Giuseppe, Triggiani Massimo, Genovese Arturo, Marone Gianni
Dipartimento di Science Mediche Traslazionali, Università degli Studi di Napoli Federico II, Via S. Pansini 5, 80131, Naples, Italy.
Department of Public Health, Università degli Studi di Napoli Federico II, Naples, Italy.
Orphanet J Rare Dis. 2016 Sep 29;11(1):133. doi: 10.1186/s13023-016-0518-8.
Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) is characterized by recurrent attacks of swelling that affect various body sites. Such attacks are a frequent cause of visits to the emergency department and are often treated in the hospital. In recent years, self-administration of C1-inhibitor (C1-INH) concentrates at home has become an increasingly used option, with a positive impact on patient outcomes and quality of life.
This was an observational study of 6 months' duration in 56 patients with C1-INH-HAE referred to a HAE center in southern Italy. The patients received three types of treatment for their swelling attacks: C1-INH concentrates administered at home (n = 25); icatibant administered at home (n = 12); and C1-INH concentrates administered in the hospital (n = 19). The objectives of this observational study were to compare therapy compliance (defined as the proportion of treated attacks) and quality of life in home- and hospital-treated patients, and to identify factors associated with the decision to use home therapy.
Overall, 918 attacks were reported over 6 months, of which 544 (59.2 %) were treated. Total number of reported attacks and the mean (±SD) number of attacks per patient, respectively, in the three groups were: 611 and 24.4 (±26.1) for home-based C1-INH; 191 and 15.9 (±12.0) for home-based icatibant; 166 and 6.1 (±6.5) for hospital-based C1-INH. Differences in attack frequency between home- and hospital-based treatments were statistically significant (p = 0.002), while patient demographic characteristics and the disease severity score did not correlate with the use of home therapy. Compliance with therapy was significantly better with home-based therapy (71.2 % of treated attacks with C1-INH and 44.0 % with icatibant) than with hospital-based therapy (21.6 %, p = 0.003). Quality of life showed an opposite trend, with patients on hospital-based treatment reporting the highest quality of life.
Home-based therapy was associated with better compliance compared with hospital-based therapy. The choice to adopt home-based therapy appeared to correlate with a high attack frequency. Home-based therapy is a valid treatment option for patients with C1-INH-HAE and should be offered to all such patients, and especially to those with high attack frequency.
C1抑制剂缺乏型遗传性血管性水肿（C1-INH-HAE）的特征是身体各部位反复出现肿胀发作。此类发作是患者频繁前往急诊科就诊的常见原因，且常在医院接受治疗。近年来，在家自行注射C1抑制剂（C1-INH）浓缩剂已成为越来越常用的一种选择，对患者的治疗效果和生活质量产生了积极影响。
这是一项针对意大利南部一家HAE中心收治的56例C1-INH-HAE患者进行的为期6个月的观察性研究。这些患者针对其肿胀发作接受了三种治疗：在家自行注射C1-INH浓缩剂（n = 25）；在家自行注射艾替班特（n = 12）；在医院注射C1-INH浓缩剂（n = 19）。这项观察性研究的目的是比较在家治疗和在医院治疗的患者的治疗依从性（定义为接受治疗的发作次数所占比例）和生活质量，并确定与选择在家治疗相关的因素。
总体而言，在6个月内共报告了918次发作，其中544次（59.2%）得到了治疗。三组中报告的发作总数及每位患者发作的平均（±标准差）次数分别为：在家自行注射C1-INH组为611次和24.4次（±26.1）；在家自行注射艾替班特组为191次和15.9次（±12.0）；在医院注射C1-INH组为166次和6.1次（±6.5）。在家治疗和在医院治疗的发作频率差异具有统计学意义（p = 0.002），而患者的人口统计学特征和疾病严重程度评分与在家治疗的选择无关。在家治疗的治疗依从性（使用C1-INH治疗的发作中有71.2%，使用艾替班特治疗的发作中有44.0%）明显优于在医院治疗（21.6%，p = 0.003）。生活质量呈现相反的趋势，在医院治疗的患者报告的生活质量最高。
与在医院治疗相比，在家治疗的依从性更好。选择在家治疗似乎与发作频率高有关。在家治疗是C1-INH-HAE患者的一种有效治疗选择，应提供给所有此类患者，尤其是发作频率高的患者。
