Abstract：
BACKGROUND: Angioedema due to acquired C1-inhibitor deficiency is a very rare but serious disease, with an estimated prevalence of 1 per 500,000 persons. There are no approved therapies to treat or prevent angioedema swelling in patients with this condition. Deucrictibant is a specific, orally bioavailable, competitive antagonist of the bradykinin B2 receptor currently under investigation for hereditary angioedema.
OBJECTIVE: Our aim was to assess the efficacy and safety of deucrictibant as acute and prophylactic treatment for angioedema due to acquired C1-inhibitor deficiency.
METHODS: A 2-part, randomized, double-blind, placebo-controlled crossover study was conducted. In Part 1, 4 consecutive angioedema attacks were treated with 3 doses of deucrictibant (10 mg, 20 mg, and 30 mg) or placebo. In Part 2, deucricibant, 20 mg, or placebo was administered twice daily for 2 treatment periods of 8 weeks.
RESULTS: Three patients were enrolled; of those 3 patients, 1 completed both study parts and 2 completed only Part 2. In Part 1, a reduction in attack severity was observed in the 3 attacks treated with deucrictibant as opposed to an increase in severity of the attack treated with placebo. In Part 2, the individual mean monthly attack rates were 2.0, 0.6, and 1.0 during the placebo period and 0.0 across all patients during treatment with deucrictibant. There were no severe adverse events and 1 self-limiting treatment-emergent adverse event (abdominal pain).
CONCLUSIONS: Deucrictibant has the potential to effectively and safely treat and prevent angioedema attacks due to acquired C1-inhibitor deficiency.
OBJECTIVE: Our aim was to assess the efficacy and safety of deucrictibant as acute and prophylactic treatment for angioedema due to acquired C1-inhibitor deficiency.
METHODS: A 2-part, randomized, double-blind, placebo-controlled crossover study was conducted. In Part 1, 4 consecutive angioedema attacks were treated with 3 doses of deucrictibant (10 mg, 20 mg, and 30 mg) or placebo. In Part 2, deucricibant, 20 mg, or placebo was administered twice daily for 2 treatment periods of 8 weeks.
RESULTS: Three patients were enrolled; of those 3 patients, 1 completed both study parts and 2 completed only Part 2. In Part 1, a reduction in attack severity was observed in the 3 attacks treated with deucrictibant as opposed to an increase in severity of the attack treated with placebo. In Part 2, the individual mean monthly attack rates were 2.0, 0.6, and 1.0 during the placebo period and 0.0 across all patients during treatment with deucrictibant. There were no severe adverse events and 1 self-limiting treatment-emergent adverse event (abdominal pain).
CONCLUSIONS: Deucrictibant has the potential to effectively and safely treat and prevent angioedema attacks due to acquired C1-inhibitor deficiency.
摘要：
背景：获得性C1抑制剂缺乏引起的血管性水肿是一种非常罕见但严重的疾病，估计患病率为每500,000人中有1人。没有批准的疗法来治疗或预防患有这种病症的患者的血管性水肿肿胀。Deucrictibant是一种特殊的，口服生物可利用性，目前正在研究的遗传性血管性水肿的缓激肽B2受体的竞争性拮抗剂。
目的：评估去红霉素作为获得性C1抑制剂缺乏所致血管性水肿的急性和预防性治疗的有效性和安全性。
方法：两部分，随机化，双盲,我们进行了安慰剂对照交叉研究.在第1部分中，用三种剂量的deucrictibant（10mg，20毫克，和30毫克）或安慰剂。在第2部分中，每天一次给药20毫克的去红霉素或安慰剂，为期8周的两个治疗期。
结果：招募了三名患者，其中一名完成了两个研究部分，两名仅完成了第二部分。在第1部分中，与用安慰剂治疗的发作严重程度增加相反，在用去酸治疗的3次发作中观察到发作严重程度降低。在第2部分中，在安慰剂期间，个体平均每月发作率为2.0、0.6和1.0，在所有患者中，在用去酸治疗期间为0.0。没有发生严重的不良事件,和一个自限性治疗出现的不良事件（腹痛）。
结论：Deucrictibant具有有效和安全地治疗和预防由于获得性C1-抑制剂缺乏引起的血管性水肿发作的潜力。
目的：评估去红霉素作为获得性C1抑制剂缺乏所致血管性水肿的急性和预防性治疗的有效性和安全性。
方法：两部分，随机化，双盲,我们进行了安慰剂对照交叉研究.在第1部分中，用三种剂量的deucrictibant（10mg，20毫克，和30毫克）或安慰剂。在第2部分中，每天一次给药20毫克的去红霉素或安慰剂，为期8周的两个治疗期。
结果：招募了三名患者，其中一名完成了两个研究部分，两名仅完成了第二部分。在第1部分中，与用安慰剂治疗的发作严重程度增加相反，在用去酸治疗的3次发作中观察到发作严重程度降低。在第2部分中，在安慰剂期间，个体平均每月发作率为2.0、0.6和1.0，在所有患者中，在用去酸治疗期间为0.0。没有发生严重的不良事件,和一个自限性治疗出现的不良事件（腹痛）。
结论：Deucrictibant具有有效和安全地治疗和预防由于获得性C1-抑制剂缺乏引起的血管性水肿发作的潜力。
