Abstract：
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.
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.
摘要：
背景：为了更好地了解遗传性血管性水肿（HAE）患者所经历的疾病的影响和负担。
目的：为了确定使用SMS与HAE患者沟通是否有助于收集发作率，药物使用和生活质量测量。
方法：邀请年龄≥12岁的医生证实的遗传性血管性水肿C1抑制剂缺乏症I型和II型患者参加。我们设计了一种新颖的方法，通过每周通过SMS使用问题来监视攻击，以实时更准确地了解澳大利亚患者的遗传性血管性水肿负担。
结果：总共向47名参与者发送了2,648条每周SMS消息，收到了1,892（71％）的回复。参与者报告了所有治疗组的463次攻击。60%的攻击得到了治疗。静脉内给予Icatibant和C1-INH浓缩物210次发作和67次发作，分别。在记录的463次袭击中，23人(5%)需要到医院就诊，主要用于面部和/或喉咙肿胀。一些参与者报告了他们选择不治疗的攻击（n=186）。大多数这些攻击的严重程度被评为轻度。21名参与者（44.7％）报告由于遗传性血管性水肿发作而损失了天数。58次攻击（17％）导致离开工作或学校的时间，相当于总时间损失85.5天。
结论：这项研究是第一次，真实世界,prospective,澳大利亚遗传性血管性水肿患者的观察性研究。尽管有有效的按需疗法，遗传性血管性水肿仍然很繁重。患有遗传性血管性水肿的患者需要更广泛地获得安全有效的预防性治疗。
目的：为了确定使用SMS与HAE患者沟通是否有助于收集发作率，药物使用和生活质量测量。
方法：邀请年龄≥12岁的医生证实的遗传性血管性水肿C1抑制剂缺乏症I型和II型患者参加。我们设计了一种新颖的方法，通过每周通过SMS使用问题来监视攻击，以实时更准确地了解澳大利亚患者的遗传性血管性水肿负担。
结果：总共向47名参与者发送了2,648条每周SMS消息，收到了1,892（71％）的回复。参与者报告了所有治疗组的463次攻击。60%的攻击得到了治疗。静脉内给予Icatibant和C1-INH浓缩物210次发作和67次发作，分别。在记录的463次袭击中，23人(5%)需要到医院就诊，主要用于面部和/或喉咙肿胀。一些参与者报告了他们选择不治疗的攻击（n=186）。大多数这些攻击的严重程度被评为轻度。21名参与者（44.7％）报告由于遗传性血管性水肿发作而损失了天数。58次攻击（17％）导致离开工作或学校的时间，相当于总时间损失85.5天。
结论：这项研究是第一次，真实世界,prospective,澳大利亚遗传性血管性水肿患者的观察性研究。尽管有有效的按需疗法，遗传性血管性水肿仍然很繁重。患有遗传性血管性水肿的患者需要更广泛地获得安全有效的预防性治疗。
