Jiménez-Yuste Víctor, Álvarez-Román María T, Berrueco Rubén, Bonanad Santiago, Calvo-Villas José M, González-González Rebeca, Porras José R González, Núñez-Vázquez Ramiro J, Rodríguez-López Manuel
Hematology Department, La Paz University Hospital-IdiPaz, Universidad Autónoma, Madrid, Spain.
Pediatric Hematology Unit, Hospital Sant Joan de Deu, Barcelona, Spain.
TH Open. 2024 Apr 17;8(2):e194-e201. doi: 10.1055/s-0044-1785525. eCollection 2024 Apr.
Management of patients with hemophilia A (HA) requires the knowledge and experience of specialized health care professionals. However, these patients may need to be attended in emergencies, outside the referral hospital, where health care professionals do not know about hemophilia and/or new innovative treatments. This study aimed to develop a simple and practical algorithm that could be used in emergency situations by nonspecialized treaters in HA and bleeding with or without factor VIII (FVIII) inhibitors under emicizumab prophylaxis. A group of experts agreed on a simple algorithm, easy to operate, adapted from previous international guidelines, and based on their clinical experience. The proposed algorithm starts with identifying the patient, confirming the diagnosis of HA, prophylaxis with emicizumab, and/or use of other treatments. After stabilizing the patient and stratifying the bleeding risk, the patient is managed according to the presence/absence of FVIII inhibitors. Patients without FVIII inhibitors should receive FVIII concentrate. Dose and follow-up depend on bleeding localization and severity. Patients with FVIII inhibitors should preferably receive recombinant activated factor VII as bypass agent. A basic coagulation assay, FVIII assessment, and FVIII inhibitors detection assays are necessary in an emergency. However, these tests should be interpreted with caution and appropriately chosen, as emicizumab may alter the results. The management of patients with HA is challenging in emergency situations, especially if they are treated with new agents. Nonspecialized in coagulopathies health care professionals have limited understanding of the disease, highlighting the need for an algorithm to assist them in making informed decisions.
甲型血友病(HA)患者的管理需要专业医护人员的知识和经验。然而,这些患者可能需要在转诊医院以外的地方接受紧急治疗,而那里的医护人员并不了解血友病和/或新的创新疗法。本研究旨在开发一种简单实用的算法,供非专业治疗人员在紧急情况下用于管理接受emicizumab预防治疗的HA患者,无论有无因子VIII(FVIII)抑制剂时的出血情况。一组专家根据他们的临床经验,在先前国际指南的基础上,商定了一种简单、易于操作的算法。所提出的算法首先要识别患者,确认HA的诊断,进行emicizumab预防治疗和/或使用其他治疗方法。在使患者病情稳定并对出血风险进行分层后,根据有无FVIII抑制剂对患者进行管理。没有FVIII抑制剂的患者应接受FVIII浓缩物治疗。剂量和随访取决于出血部位和严重程度。有FVIII抑制剂的患者最好接受重组活化因子VII作为旁路药物治疗。紧急情况下需要进行基本凝血检测、FVIII评估和FVIII抑制剂检测。然而,由于emicizumab可能会改变检测结果,因此对这些检测结果的解读应谨慎且选择恰当。在紧急情况下,HA患者的管理具有挑战性,尤其是在使用新药物治疗时。非凝血疾病专业的医护人员对该疾病的了解有限,这凸显了需要一种算法来帮助他们做出明智的决策。