CSL Behring, King of Prussia, PA, USA.
AARA Research Center, Dallas, TX, USA.
Orphanet J Rare Dis. 2024 Jun 22;19(1):241. doi: 10.1186/s13023-024-03247-1.
Hereditary angioedema (HAE) is characterized by unpredictable and often severe cutaneous and mucosal swelling that affects the extremities, face, larynx, gastrointestinal tract, or genitourinary area. Introduction of novel long-term prophylactic treatment options (lanadelumab, berotralstat, and C1-esterase inhibitor SC [human]) into the treatment armamentarium has substantially reduced HAE attacks, allowing patients to be attack free for longer with improvements to their quality of life. Using data drawn from a wide-ranging survey of patients with HAE, we examined the relationship between duration of time attack free and health-related quality of life (HRQoL), exploring the possibility that there is an association between observed improvement in HRQoL and attack-free duration.
A survey among patients with HAE on long-term prophylaxis (LTP) in six countries (the US, Australia, Canada, UK, Germany, and Japan) assessed the relationship between attack-free duration and mean Angioedema Quality of Life (AE-QoL) scores, quality of life benefits, and rescue medication used. Analysis of covariance (ANCOVA) was used to assess the roles of LTP and attack-free period (< 1 month, 1- < 6 months, ≥ 6 months) on total AE-QoL scores. Results include descriptive p-values for strength of association, without control for multiplicity. Descriptive statistics were used to show the relationship between time attack free and quality of life benefits.
Longer durations of time for which participants reported being attack free at the time of the survey correlated with better AE-QoL scores and less use of rescue medication. The mean total AE-QoL scores were 51.8, 33.2, and 19.9 for those who reported having been attack free for < 1 month, 1- < 6 months, and ≥ 6 months, respectively, with higher scores reflecting more impairment. The ANCOVA results showed a strong association between attack-free duration and AE-QoL total score.
This study shows that longer attack-free duration has an influential role for better HRQoL in patients receiving LTP. Prolonging the attack-free period is an important goal of therapy and recent advances in LTP have increased attack-free duration. However, opportunities exist for new treatments to further increase attack-free duration and improve HRQoL for all patients with HAE.
遗传性血管性水肿 (HAE) 的特征是皮肤和黏膜出现不可预测且常为严重的肿胀，影响四肢、面部、喉部、胃肠道或泌尿生殖区。新型长期预防治疗选择（拉那芦单抗、贝曲西班和 C1 酯酶抑制剂 SC[人源]）的引入极大地减少了 HAE 发作，使患者能够更长时间地免于发作，生活质量得到改善。利用一项对 HAE 患者进行的广泛调查数据，我们研究了免于发作时间与健康相关生活质量 (HRQoL) 之间的关系，探索了观察到的 HRQoL 改善与无发作时间之间是否存在关联。
在六个国家（美国、澳大利亚、加拿大、英国、德国和日本）对接受长期预防治疗 (LTP) 的 HAE 患者进行了一项调查，评估了免于发作时间与血管性水肿生活质量 (AE-QoL) 评分、生活质量获益和使用的急救药物之间的关系。使用协方差分析 (ANCOVA) 评估了 LTP 和无发作期 (<1 个月、1-<6 个月、≥6 个月) 对总 AE-QoL 评分的作用。分析结果包括关联强度的描述性 p 值，无需对多重性进行控制。使用描述性统计来显示无发作时间与生活质量获益之间的关系。
参与者在调查时报告的无发作时间越长，AE-QoL 评分越高，急救药物的使用越少。报告无发作时间<1 个月、1-<6 个月和≥6 个月的患者的平均总 AE-QoL 评分分别为 51.8、33.2 和 19.9，评分越高表明损害越大。ANCOVA 结果表明，无发作时间与 AE-QoL 总分之间存在强烈关联。
这项研究表明，在接受 LTP 的患者中，更长的无发作时间与更好的 HRQoL 相关。延长无发作时间是治疗的重要目标，LTP 的最新进展已延长了无发作时间。然而，仍有机会利用新的治疗方法进一步延长所有 HAE 患者的无发作时间并改善 HRQoL。
