Rixubis获准用于治疗B型血友病患者

圣路易斯(MD Consult)——2013年6月27日，百特公司宣布，美国食品药品管理局(FDA)已批准Rixubis［凝血因子Ⅸ（重组）］用于成年B型血友病患者的常规预防性治疗、出血发作控制和术前管理。B型血友病是第二常见的血友病类型，是血液中自然产生的一种控制出血的蛋白质——凝血因子Ⅸ数量不足造成的。据百特公司介绍，Rixubis是唯一一种同时囊括对此类患者的常规预防和出血发作控制适应证的重组因子Ⅸ。

这一批准令是基于一项Ⅰ/Ⅲ期研究的数据。该研究结果显示，每周2次使用Rixubis预防性治疗6个月，中位年出血率可达到2.0，43%的患者未发生出血。在这项研究中，无一患者产生对因子Ⅸ的抑制性抗体，且未报告有过敏病例。

临床研究中最常见的不良反应为味觉障碍、四肢疼痛和furin抗体阳性。

使用含因子Ⅸ的产品时，曾报告有发生超敏反应（包括过敏）的病例。早期过敏反应体征可进展为过敏，包括血管神经系统水肿、胸部压迫感、低血压、嗜睡、恶心、呕吐、感觉异常、烦躁不安、喘息和呼吸困难。

接受Rixubis治疗的患者可能产生对因子Ⅸ的中和抗体（抑制剂）。应通过适当的临床观察和实验室检测，定期评估患者是否产生因子Ⅸ抑制剂。如在使用预期剂量的情况下未达到预期的因子IX血浆活性水平，或出血未能控制，应进行测定因子Ⅸ抑制剂浓度的分析。产生因子Ⅸ抑制剂的患者如再次暴露于Rixubis，发生重度超敏反应的风险增高。

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ST LOUIS (MD Consult) - On June 27, 2013, Baxter announced that the US Food and Drug Administration (FDA) has approved Rixubis (coagulation factor IX [recombinant]) for routine prophylactic treatment, control of bleeding episodes, and perioperative management in adults with hemophilia B. Hemophilia B is the second most common type of hemophilia and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding. According to Baxter, Rixubis is the only recombinant factor IX indicated for both routine prophylaxis and control of bleeding episodes in such patients.

This approval was granted on the basis of data from a phase I/III study. Results of the study demonstrated that twice-weekly prophylactic treatment with Rixubis for 6 months achieved a median annualized bleed rate of 2.0, with 43% of patients experiencing no bleeds. In this study, no patients showed development of an inhibitory antibody to factor IX and no cases of anaphylaxis were reported.

The most common adverse reactions observed in clinical studies were dysgeusia, extremity pain, and positive furin antibody testing.

Hypersensitivity reactions, including anaphylaxis, have been reported with the use of factor IX-containing products. Early signs of allergic reactions, which can progress to anaphylaxis, include angioedema, chest tightness, hypotension, lethargy, nausea, vomiting, paresthesia, restlessness, wheezing, and dyspnea.

Development of neutralizing antibodies (inhibitors) to factor IX may occur in patients receiving Rixubis. Patients should be regularly evaluated for the development of factor IX inhibitors by appropriate clinical observations and laboratory tests. If expected factor IX plasma activity levels are not attained, or if bleeding is not controlled with an expected dose, an assay should be performed that measures factor IX inhibitor concentration. Patients with factor IX inhibitors are at an increased risk of severe hypersensitivity reactions or anaphylaxis if re-exposed to Rixubis.

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