Abstract：
BACKGROUND: Hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) is a rare disease characterized by swelling episodes. It affects quality of life (QOL) and can be fatal when the upper airways are involved. Treatment is individualized, with therapeutic options including on-demand treatment (ODT) and short- and long-term prophylaxis (STP, LTP). However, available guidelines are not always clear about the selection of treatment, the goals of treatment, or how achievement of these goals is assessed.
OBJECTIVE: To review available evidence for the management of HAE-C1INH and build a Spanish expert consensus to steer management towards a treat-to-target approach, while addressing some of the less clear aspects of the Spanish guidelines.
METHODS: We reviewed the literature on the treat-to-target management of HAE-C1INH, focusing on treatment selection and goals and the tools available to assess whether the goals have been achieved. We discussed the literature based on clinical experience and drew up 45 statements on undefined management aspects. A panel of 53 HAE experts validated the statements through a 2-round Delphi process.
RESULTS: The goals for ODT and STP are to minimize the morbidity and mortality of attacks and to prevent attacks caused by known triggers, respectively, while the main goal of LTP is to decrease the rate, severity, and duration of attacks. Furthermore, when prescribing, clinicians should consider the reduction in adverse effects, while increasing patient QOL and satisfaction. Appropriate instruments for assessing achievement of treatment goals are also indicated.
CONCLUSIONS: We provide recommendations on previously unclear aspects of HAE-C1INH management with ODT, STP, and LTP, focusing on clinical and patient-oriented goals.
OBJECTIVE: To review available evidence for the management of HAE-C1INH and build a Spanish expert consensus to steer management towards a treat-to-target approach, while addressing some of the less clear aspects of the Spanish guidelines.
METHODS: We reviewed the literature on the treat-to-target management of HAE-C1INH, focusing on treatment selection and goals and the tools available to assess whether the goals have been achieved. We discussed the literature based on clinical experience and drew up 45 statements on undefined management aspects. A panel of 53 HAE experts validated the statements through a 2-round Delphi process.
RESULTS: The goals for ODT and STP are to minimize the morbidity and mortality of attacks and to prevent attacks caused by known triggers, respectively, while the main goal of LTP is to decrease the rate, severity, and duration of attacks. Furthermore, when prescribing, clinicians should consider the reduction in adverse effects, while increasing patient QOL and satisfaction. Appropriate instruments for assessing achievement of treatment goals are also indicated.
CONCLUSIONS: We provide recommendations on previously unclear aspects of HAE-C1INH management with ODT, STP, and LTP, focusing on clinical and patient-oriented goals.
摘要：
背景：伴有C1抑制剂缺乏症的遗传性血管性水肿（HAE-C1INH）是一种以肿胀发作为特征的罕见疾病。它影响生活质量（QoL）并且可能致命，当涉及上呼吸道时。治疗是个性化的，治疗方案包括按需治疗（ODT），以及短期和长期预防(STP，LTP)。然而,可用的指南并不总是明确的治疗选择，其目标或目标成就的评估。
目的：审查HAE-C1INH管理的现有证据，并建立西班牙专家共识，以指导HAE-C1INH管理朝着治疗目标（T2T）方法发展，同时解决了西班牙指南中一些不清楚的方面。
方法：我们回顾了用T2T方法管理HAE-C1INH的文献，重点关注：1)治疗选择和目标；2)评估目标成就的可用工具。我们根据临床经验讨论了文献，并就未定义的管理方面起草了45份声明。由53名HAE专家组成的小组通过两轮Delphi流程验证了这些陈述。
结果：ODT和STP的目标是将发作的发病率和死亡率降至最低，并防止由已知触发器引起的攻击，分别,虽然LTP的主要目标是降低利率，攻击的严重程度和持续时间。此外，开处方时，临床医生应该考虑减少不良事件，同时增加患者QoL和满意度。还指出了评估目标成就的适当工具。
结论：我们就ODT治疗HAE-C1INH之前不清楚的方面提供建议，STP和LTP，特别关注临床和面向患者的目标。
目的：审查HAE-C1INH管理的现有证据，并建立西班牙专家共识，以指导HAE-C1INH管理朝着治疗目标（T2T）方法发展，
方法：我们回顾了用T2T方法管理HAE-C1INH的文献，
结果：ODT和STP的目标是将发作的发病率和死亡率降至最低，
结论：我们就ODT治疗HAE-C1INH之前不清楚的方面提供建议，
