López-Jaime Francisco-José, Benítez Olga, Díaz Jordán Bolívar Luis, Montaño Adrián, Coll Julia, Quintana París Laura, Gómez-Del Castillo Solano María Del Carmen
Unidad de Hemostasia y Trombosis, Hospital Universitario Regional de Málaga, IBIMA, Málaga, Spain.
Departamento de Hematología Experimental, VHIO Vall d'Hebron Instituto de Oncología, Hospital Universitario Vall d'Hebron, Barcelona, Spain.
Hematology. 2023 Dec;28(1):2166334. doi: 10.1080/16078454.2023.2166334.
Prophylaxis with emicizumab, a bispecific monoclonal antibody that mimics FVIII function, has shown encouraging results in clinical trials in terms of efficacy and safety. However, current experience is limited, and many areas of concern to clinicians have yet to be reviewed.
This paper reviews the experience of hemophilia A patients treated with emicizumab based on the results of clinical trials and real-life studies. The authors place special emphasis on issues such as the management of these patients in situations of hemorrhage and/or surgical interventions, joint health or laboratory monitoring.
Treatment with emicizumab has been shown to improve joint health and reduce bleeding, of particular interest to patients with inhibitors and high bleeding rates. However, there are still concerns about its administration in neonates and previously untreated patients due to limited reported experience. Laboratory monitoring is not strictly necessary due to the stable pharmacokinetics emicizumab has been shown to exhibit, however, tests that globally assess hemostasis may be useful especially in cases of bleeding or surgery. The authors are also of the opinion that prophylaxis before minor surgery is not necessary and that major surgeries can be safely performed with additional prophylactic coagulation factor. ClinicalTrials.gov identifier: NCT04431726..
emicizumab是一种模拟FVIII功能的双特异性单克隆抗体，其预防性治疗在临床试验中已显示出令人鼓舞的疗效和安全性结果。然而，目前的经验有限，临床医生关注的许多领域尚未得到审查。
本文基于临床试验和实际研究结果，综述了接受emicizumab治疗的A型血友病患者的经验。作者特别强调了这些患者在出血和/或手术干预情况下的管理、关节健康或实验室监测等问题。
已证明emicizumab治疗可改善关节健康并减少出血，这对有抑制物和高出血率的患者尤为重要。然而，由于报告的经验有限，对于新生儿和既往未治疗的患者使用该药物仍存在担忧。由于已证明emicizumab具有稳定的药代动力学，因此实验室监测并非严格必要，但是，全面评估止血功能的检测可能会有用，尤其是在出血或手术情况下。作者还认为，小手术前无需进行预防性治疗，大手术可通过额外的预防性凝血因子安全进行。ClinicalTrials.gov标识符：NCT04431726。
