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Abstract：
BACKGROUND: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, recurring subcutaneous or submucosal swelling. Without effective therapy, HAE can negatively impact patients\' quality of life. Management of HAE includes on-demand treatment of attacks and short- and long-term prophylaxis (LTP) to prevent attacks. Newer therapies may be more tolerable and effective in managing HAE; however, therapies such as androgens are still widely used in some countries owing to their relative ease of access and adequate disease control for some patients. This study evaluated the characteristics, treatment patterns, clinical outcomes, and healthcare resource utilization of a multinational cohort of patients with HAE, with a focus on understanding reasons for recommending or discontinuing available therapies.
METHODS: A retrospective chart review was conducted at 12 centers in six countries and included data from patients with HAE type 1 or 2 who were ≥ 12 years of age at their first clinical visit. The relationship between LTP use and attack rates was evaluated using a multivariable Poisson regression model. Data were collected between March 2018 and July 2019.
RESULTS: Data from 225 patients were collected (62.7% female, 86.2% White, 90.2% type 1); 64.4% of patients had their first HAE-related visit to the center prior to or during 2014. Treatment patterns varied between countries. Overall, 85.8% of patients were prescribed on-demand treatment and 53.8% were prescribed LTP, most commonly the androgen danazol (53.7% of patients who used LTP). Plasma-derived C1 inhibitor (Cinryze®) was used by 29.8% of patients for LTP. Patients who received LTP had a significantly lower rate of HAE attacks than patients who did not receive any LTP (incidence rate ratio (95% confidence interval) 0.90 (0.84-0.96)). Androgens were the most commonly discontinued therapy (51.3%), with low tolerability cited as the most frequent reason for discontinuation (50.0%).
CONCLUSIONS: Overall, findings from this study support the use of LTP in the prevention of HAE attacks; a lower rate of attacks was observed with LTP compared with no LTP. However, the type of LTP used varied between countries, with tolerability and accessibility to specific treatments playing important roles in management decision-making.
METHODS: A retrospective chart review was conducted at 12 centers in six countries and included data from patients with HAE type 1 or 2 who were ≥ 12 years of age at their first clinical visit. The relationship between LTP use and attack rates was evaluated using a multivariable Poisson regression model. Data were collected between March 2018 and July 2019.
RESULTS: Data from 225 patients were collected (62.7% female, 86.2% White, 90.2% type 1); 64.4% of patients had their first HAE-related visit to the center prior to or during 2014. Treatment patterns varied between countries. Overall, 85.8% of patients were prescribed on-demand treatment and 53.8% were prescribed LTP, most commonly the androgen danazol (53.7% of patients who used LTP). Plasma-derived C1 inhibitor (Cinryze®) was used by 29.8% of patients for LTP. Patients who received LTP had a significantly lower rate of HAE attacks than patients who did not receive any LTP (incidence rate ratio (95% confidence interval) 0.90 (0.84-0.96)). Androgens were the most commonly discontinued therapy (51.3%), with low tolerability cited as the most frequent reason for discontinuation (50.0%).
CONCLUSIONS: Overall, findings from this study support the use of LTP in the prevention of HAE attacks; a lower rate of attacks was observed with LTP compared with no LTP. However, the type of LTP used varied between countries, with tolerability and accessibility to specific treatments playing important roles in management decision-making.
摘要：
背景：遗传性血管性水肿（HAE）是一种罕见的疾病，其特征是不可预测的，复发性皮下或粘膜下肿胀。没有有效的治疗,HAE会对患者的生活质量产生负面影响。HAE的管理包括按需治疗发作和短期和长期预防(LTP)以防止发作。较新的疗法可能在管理HAE方面更具耐受性和有效性；然而，由于雄激素等疗法相对容易获得，并且对某些患者进行了充分的疾病控制，因此在一些国家仍然广泛使用。这项研究评估了这些特征，治疗模式,临床结果，以及一个多国HAE患者队列的医疗资源利用情况，重点了解推荐或停止可用疗法的原因。
方法：在6个国家的12个中心进行了回顾性分析，纳入了首次临床就诊时年龄≥12岁的1型或2型HAE患者的数据。使用多变量Poisson回归模型评估LTP使用率与发作率之间的关系。数据收集时间为2018年3月至2019年7月。
结果：收集了225例患者的数据（62.7％为女性，86.2%白色，90.2%的1型）；64.4%的患者在2014年之前或期间首次访问该中心。各国的治疗模式各不相同。总的来说,85.8%的患者进行了按需治疗，53.8%的患者进行了LTP，最常见的是雄激素达那唑(53.7%的患者使用LTP)。29.8%的患者使用血浆衍生的C1抑制剂(Cinryze®)治疗LTP。与未接受任何LTP的患者相比，接受LTP的患者的HAE发作率明显较低（发生率比（95％置信区间）0.90（0.84-0.96））。雄激素是最常见的停药治疗（51.3%），耐受性低被认为是停药的最常见原因(50.0%)。
结论：总体而言，这项研究的结果支持使用LTP预防HAE发作;与没有LTP相比,使用LTP观察到较低的发作率.然而,使用的LTP类型因国家而异，对特定治疗的耐受性和可及性在管理决策中发挥着重要作用。
方法：在6个国家的12个中心进行了回顾性分析，纳入了首次临床就诊时年龄≥12岁的1型或2型HAE患者的数据。使用多变量Poisson回归模型评估LTP使用率与发作率之间的关系。数据收集时间为2018年3月至2019年7月。
结果：收集了225例患者的数据（62.7％为女性，86.2%白色，90.2%的1型）；64.4%的患者在2014年之前或期间首次访问该中心。各国的治疗模式各不相同。总的来说,85.8%的患者进行了按需治疗，53.8%的患者进行了LTP，最常见的是雄激素达那唑(53.7%的患者使用LTP)。29.8%的患者使用血浆衍生的C1抑制剂(Cinryze®)治疗LTP。与未接受任何LTP的患者相比，接受LTP的患者的HAE发作率明显较低（发生率比（95％置信区间）0.90（0.84-0.96））。雄激素是最常见的停药治疗（51.3%），耐受性低被认为是停药的最常见原因(50.0%)。
结论：总体而言，这项研究的结果支持使用LTP预防HAE发作;与没有LTP相比,使用LTP观察到较低的发作率.然而,使用的LTP类型因国家而异，对特定治疗的耐受性和可及性在管理决策中发挥着重要作用。
