背景:遗传性血管性水肿(HAE)的特征是不可预测的,通常是严重的皮肤和粘膜肿胀,影响四肢,脸,喉部,胃肠道,或泌尿生殖系统。引入新的长期预防性治疗方案(lanadelumab,berotralstat,和C1酯酶抑制剂SC[人])进入治疗设备已经大大减少了HAE攻击,使患者可以更长时间地自由发作,从而改善他们的生活质量。使用从HAE患者的广泛调查中得出的数据,我们检查了无攻击持续时间与健康相关生活质量(HRQoL)之间的关系,探索观察到的HRQoL改善与无攻击持续时间之间存在关联的可能性。
方法:在六个国家(美国,澳大利亚,加拿大,英国,德国,和日本)评估了无发作持续时间与平均血管性水肿生活质量(AE-QoL)评分之间的关系,生活质量效益,和使用救援药物。协方差分析(ANCOVA)用于评估LTP和无发作期(<1个月,1-<6个月,≥6个月)的总AE-QoL评分。结果包括关联强度的描述性p值,不控制多重性。描述性统计用于显示无时间攻击与生活质量之间的关系。
结果:参与者在调查时报告无发作的持续时间较长,与较好的AE-QoL评分和较少使用救援药物相关。报告无发作时间<1个月的患者的平均总AE-QoL评分分别为51.8、33.2和19.9。1-<6个月,≥6个月,分别,分数越高,反映出更多的损害。ANCOVA结果显示无攻击持续时间和AE-QoL总分之间有很强的相关性。
结论:这项研究表明,在接受LTP的患者中,较长的无发作持续时间对较好的HRQoL具有影响作用。延长无发作期是治疗的重要目标,LTP的最新进展增加了无发作期。然而,对于所有HAE患者,存在进一步延长无发作持续时间和改善HRQoL的新疗法的机会.
BACKGROUND: 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.
METHODS: 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.
RESULTS: 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.
CONCLUSIONS: 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.