Department of Pediatrics, Nara Medical University, Kashihara, Nara, Japan.
Thrombosis and Hemostasis Research Centre, Nara Medical University, Kashihara, Nara, Japan.
Br J Haematol. 2023 Jan;200(1):23-34. doi: 10.1111/bjh.18379. Epub 2022 Jul 23.
Some non-factor products that work by facilitating the coagulation pathway (emicizumab) and blocking the anticoagulant pathway (fitusiran, concizumab and marstacimab) for patients with haemophilia (H) have been developed, and clinical trials using these products are currently ongoing. Prophylaxis using non-factor products by subcutaneous administration provides marked reductions of bleeding episodes in patients with HA or HB, regardless of the presence of inhibitor. Emicizumab has already been approved globally. Emicizumab alters the phenotype of patients with HA from severe to mild by maintaining trough levels of equivalent factor VIII activity (15-20 iu/dl). Phase 3 clinical trials and long-term observations assessing emicizumab revealed tolerable safety and efficacy. However, thrombotic events have occurred in patients receiving these non-factor products. Furthermore, monitoring of the haemostatic function of these products with concomitant therapy is also required in clinical practice. These products have promising haemostatic efficiency, but wider clinical experience is needed to provide optimal therapeutic strategies in the future.
一些通过促进凝血途径（依库珠单抗）和阻断抗凝途径（fitusiran、concizumab 和 marstacimab）治疗血友病（H）的非因子产品已经开发出来，并且目前正在使用这些产品进行临床试验。通过皮下给药使用非因子产品进行预防，无论是否存在抑制剂，均可显著减少 HA 或 HB 患者的出血发作次数。依库珠单抗已经在全球获得批准。依库珠单抗通过维持相当于因子 VIII 活性的谷浓度（15-20 iu/dl），将 HA 患者的表型从严重改变为轻度。评估依库珠单抗的 3 期临床试验和长期观察结果显示其具有可耐受的安全性和疗效。然而，接受这些非因子产品治疗的患者发生了血栓事件。此外，在临床实践中还需要同时治疗监测这些产品的止血功能。这些产品具有有前途的止血效率，但需要更广泛的临床经验来提供未来的最佳治疗策略。
