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2022 年在资源有限、半衰期延长的浓缩物或非替代疗法的情况下管理血友病患者的侵入性操作。

Managing invasive procedures in haemophilia patients with limited resources, extended half-life concentrates or non-replacement therapies in 2022.

机构信息

Center for Thrombosis and Haemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.

Sahyadri Speciality Hospital, Pune, Maharashtra, India.

出版信息

Haemophilia. 2022 May;28 Suppl 4:93-102. doi: 10.1111/hae.14551.

Abstract

New treatment possibilities and modalities are now available globally for patients with haemophilia requiring surgery or invasive procedures. The first is the appropriate application of low-dose protocols of clotting factor concentrates (CFC) achieving adequate perioperative haemostasis in resources constraint environments. The increasing availability of CFC through humanitarian aid programs allows more invasive surgeries to be performed for which efficacy and safety data should be more widely collected and reported. Second, extended half-life CFC that are increasingly available in many countries represent valuable alternatives to standard half-life products in surgical patients allowing reduced number of infusions and lower consumption, in particular for extended half-life factor IX. Third, in the era of recently introduced nonfactor prophylaxis, some minor surgical procedures can now be performed without additional haemostatic treatment, others with few low-dose administrations of CFC or bypassing agents. Additional factor VIII/IX or recombinant activated factor VII has proven to be safe and effective in association with emicizumab for major surgeries and it was effectively given at low doses in association with fitusiran (including activated prothrombin complex concentrate). No thrombotic complications have been reported in the surgical setting so far. A multidisciplinary team/facility remains crucial to manage major surgery in patients on prophylaxis with these new agents.

摘要

目前,全球范围内需要接受手术或有创操作的血友病患者可获得新的治疗选择和方案。首先,在资源有限的环境中,通过适当应用低剂量凝血因子浓缩物(CFC)方案,可以实现围手术期充分止血。通过人道主义援助计划越来越多地提供 CFC,使得更多的侵袭性手术得以进行,应该更广泛地收集和报告这些手术的疗效和安全性数据。其次,在许多国家,越来越多地可获得半衰期延长的 CFC,这为手术患者提供了有价值的标准半衰期产品替代方案,可减少输注次数和降低消耗,尤其是对于半衰期延长的因子 IX。第三,在最近推出非因子预防性治疗的时代,一些小型手术现在可以无需额外的止血治疗进行,而其他手术则可以使用少量低剂量的 CFC 或旁路制剂进行。在接受emicizumab 联合治疗的大型手术中,添加的因子 VIII/IX 或重组激活因子 VII 已被证明是安全有效的,并且在与 fitusiran(包括激活的凝血酶原复合物浓缩物)联合使用时,也可以以低剂量有效给药。迄今为止,在手术环境中尚未报告血栓并发症。对于使用这些新型药物进行预防性治疗的患者,多学科团队/机构对于管理大型手术仍然至关重要。

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