Immunology and Allergy Unit, Campbelltown Hospital, Campbelltown, New South Wales, Australia; Faculty of Medicine, Western Sydney University, Campbelltown, New South Wales, Australia.
Aesir Health, Cheltenham, Victoria, Australia.
J Allergy Clin Immunol Pract. 2023 Aug;11(8):2457-2467.e1. doi: 10.1016/j.jaip.2023.02.037. Epub 2023 Mar 12.
To understand the impact and burden of disease experienced by patients with hereditary angioedema (HAE).
To determine whether the use of short message service (SMS) to communicate with patients with HAE facilitates the collection of attack rate, medication use, and quality of life measurements.
Patients aged 12 years and older with doctor-confirmed HAE C1-inhibitor deficiency types I and II were invited to participate. We devised a novel method for monitoring attacks by using questions weekly via SMS to gain a more accurate picture of the burden of HAE in Australian patients in real time.
A total of 2,648 weekly SMS messages were sent to 47 participants; 1,892 responses were received (71%). Participants reported 463 attacks across all treatment groups. Sixty percent of attacks were treated. Icatibant and C1-inhibitor concentrate were administered IV for 210 and 67 attacks, respectively. Of the 463 recorded attacks, 23 necessitated presentation to the hospital (5%), predominantly for facial and/or throat swelling. Several participants reported attacks (n = 186), which they chose not to treat. Most of those attacks were rated mildly severe. Twenty-one participants reported lost days owing to HAE attacks (44.7%). Fifty-eight attacks (17%) resulted in time away from work or school, equating to a total of 85.5 days lost.
This study was a first of its kind, real-world, prospective, observational study of Australian patients living with HAE. Despite the availability of effective on-demand therapies, HAE remains burdensome. Wider access to safe and effective prophylactic therapies is needed for patients living with HAE.
了解遗传性血管性水肿（HAE）患者所经历的疾病影响和负担。
确定使用短消息服务（SMS）与 HAE 患者进行通信是否有助于收集发作率、药物使用和生活质量测量值。
邀请年龄在 12 岁及以上、经医生确诊为 C1 抑制剂缺乏型 I 型和 II 型 HAE 的患者参与。我们设计了一种新的监测发作的方法，通过每周通过 SMS 向患者发送问题，实时更准确地了解澳大利亚 HAE 患者的负担情况。
共向 47 名参与者发送了 2648 条每周 SMS 消息，收到了 1892 条回复（71%）。所有治疗组的参与者报告了 463 次发作。60%的发作得到了治疗。伊卡替班和 C1 抑制剂浓缩物分别用于 210 次和 67 次发作的 IV 治疗。在记录的 463 次发作中，有 23 次需要到医院就诊（5%），主要是因为面部和/或喉咙肿胀。一些参与者报告了发作（n=186），但他们选择不治疗。这些发作大多数被评为轻度严重。21 名参与者报告因 HAE 发作而失去了天数（44.7%）。58 次发作（17%）导致离开工作或学校，总共损失了 85.5 天。
这是一项首例对澳大利亚 HAE 患者进行的真实世界、前瞻性、观察性研究。尽管有有效的按需治疗方法，但 HAE 仍然是一种负担。需要为 HAE 患者提供更广泛的安全有效的预防治疗方法。
