ST LOUIS (MD Consult) - On October 12, 2009, CSL Behring announced that the US food and Drug Administration has approved Berinert for the treatment of acute abdominal or facial manifestations of hereditary angioedema (HAE) in adults and adolescents. At the time of this approval, Berinert became the first therapy approved for this indication in the United States.
According to the manufacturer's press release, HAE is a genetic disorder caused by a deficiency of the C1 inhibitor (C1-INH). The disorder is inherited in an autosomal dominant manner. Symptoms of HAE include episodes of edema or swelling in the face and the abdomen. Patients who are subject to abdominal manifestations of HAE can experience episodes of severe pain, diarrhea, nausea, and vomiting as a result of intestinal wall swelling. Episodes of HAE that involve the face can cause painful distortion and swelling.
Berinert, a plasma-derived intravenous therapy, is used to treat the fundamental cause of HAE symptoms by providing patients who have C1-INH deficiency with the human protein that is missing from their systems.
The approval of Berinert for the treatment of HAE was granted on the basis of results from a prospective, phase 2/3, international, multicenter, double-blind, placebo-controlled trial in which the efficacy and safety of C1-INH concentrate were studied. The trial involved 124 patients who were experiencing moderate or severe acute abdominal or facial manifestations of HAE. In trial participants, the median time to symptom relief was 30 minutes after receiving C1-INH, compared with 1.5 hours after receipt of placebo.
The most serious adverse reaction reported by participants in clinical studies who received Berinert was an increase in the severity of pain associated with HAE. The most common adverse reactions observed in >4% of patients after receiving Berinert treatments were headache, abdominal pain, nausea, muscle spasms, pain, diarrhea, and vomiting.
Berinert is derived from human plasma. The risk of transmission of infectious agents including viruses and, theoretically, the Creutzfeldt-Jakob disease agent, cannot be completely eliminated.
Berinert is contraindicated in persons who have experienced an anaphylactic or severe systemic reaction to C1-INH preparations. Patients should be monitored for early signs of allergic or hypersensitivity reactions (including hives, generalized urticaria, chest tightness, wheezing, hypotension, and anaphylaxis). If hypersensitivity is suspected, the Berinert infusion should be immediately discontinued and appropriate treatment initiated. Epinephrine should be immediately available for the treatment of acute severe hypersensitivity reactions.
The safety and efficacy of Berinert for the prophylaxis of HAE have not been established.
Copyright (c) 2009 Elsevier Global Medical News. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
圣路易斯(MD Consult)——2009年10月12日，CSL Behring公司宣布，美国食品药品管理局(FDA)已批准Berinert作为成人及青少年遗传性血管性水肿(HAE)腹部或面部急性发作的对症治疗药。批准之时，Berinert已成为美国已获准的针对该适应证的一线治疗药。
据生产厂家召开的新闻发布会声称，HAE是一种由C1抑制剂(C1-INH)缺乏所致的遗传疾病。该疾病的遗传方式为常染色体显性遗传。HAE的症状包括面部和腹部发生水肿或肿胀。HAE累及腹部时可出现腹部剧痛、腹泻、恶心以及呕吐等症状，此系肠壁肿胀所致；累及面部时可引起面部疼痛性扭曲及肿胀。
Berinert是一种血浆源性静脉治疗药，通过为C1-INH缺乏患者提供其系统中所缺乏的蛋白，治疗产生HAE症状的基本病因。
FDA批准该药用于治疗HAE是基于一项前瞻性、II/III期、国际、多中心、双盲、安慰剂对照试验的结果，该研究对C1-INH的疗效和安全性进行了探讨。试验纳入了124例HAE中或重度急性腹部或面部发作的患者。在参与者中，接受C1-INH治疗组患者症状缓解的中位时间为30 min，而安慰剂对照组为1.5 h。
临床研究中接受Berinert治疗的患者所报告的最严重不良反应，为HAE相关疼痛的严重程度增加。接受该药治疗后患者最常见(即>4%在接受治疗后出现)的不良反应为头痛、腹痛、恶心、肌肉痉挛、疼痛、腹泻以及呕吐。
Berinert源于人血浆。病毒以及理论上克罗伊茨费尔特-雅各布病因子等传染性因子的传播风险无法彻底消除。
Berinert禁用于已发生对C1-INH制剂过敏或重度全身反应的患者。应对患者进行变态反应或超敏反应的早期体征(包括荨麻疹、全身风疹、胸闷、喘鸣、低血压以及过敏反应)监测。若疑似超敏反应，应即刻停止输注Beriner并进行适当治疗。应立即使用肾上腺素治疗急性重度超敏反应。
至今尚未证实Berinert预防HAE的安全性和效果。
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