Background

Idiopathic anaphylaxis (IA) is a diagnosis of exclusion, thus taking away the option of therapeutic management focused on eliminating the inciting agent. Epinephrine and antihistamines followed by systemic corticosteroids are the mainstays of therapy for acute events. There is no prophylactic therapy that reliably prevents anaphylaxis.

Objective

We sought to determine the efficacy of omalizumab in the management of patients with frequent episodes of IA in a double-blind, placebo-controlled trial.

Methods

We prospectively enrolled 19 patients with frequent IA (≥6 episodes/y) who then underwent a medical evaluation that included a serum tryptase determination, mutational analysis for KIT D816V, and bone marrow evaluation to rule out a clonal mast cell disorder. Computer-generated random numbers were provided by the study pharmacist. The primary end point was anaphylactic events in the 6 months after baseline. Sixteen patients completed the primary trial.

Results

No statistically significant difference was demonstrated between the placebo and treated groups. There was a trend for efficacy in the treatment group, particularly after 60 days. Overall, the safety profile was favorable without long-term side effects.

Conclusions

Omalizumab was safely administered to a difficult-to-treat patient population with IA. The efficacy results trended modestly in favor of the treatment group, but no statistically significant differences were detected.

中文翻译：

---

奥马珠单抗治疗特发性过敏反应的随机双盲、安慰剂对照研究

背景

特发性过敏反应 (IA) 是一种排除性诊断，因此取消了专注于消除诱发因素的治疗管理选择。肾上腺素和抗组胺药，然后是全身性皮质类固醇是急性事件的主要治疗方法。没有预防性治疗可以可靠地防止过敏反应。

客观的

我们试图在一项双盲、安慰剂对照试验中确定奥马珠单抗在治疗频繁发作的 IA 患者中的疗效。

方法

我们前瞻性地招募了 19 名频繁 IA（≥6 次/年）的患者，然后他们接受了医学评估，包括血清类胰蛋白酶测定、KIT D816V突变分析和骨髓评估，以排除克隆性肥大细胞疾病。计算机生成的随机数由研究药剂师提供。主要终点是基线后 6 个月内的过敏事件。16 名患者完成了初步试验。

结果

安慰剂组和治疗组之间没有显示出统计学上的显着差异。治疗组有疗效的趋势，特别是在 60 天后。总体而言，安全性良好，没有长期副作用。

结论

Omalizumab 被安全地用于难以治疗的 IA 患者群体。疗效结果略有利于治疗组，但未检测到统计学上的显着差异。