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.
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。