Abstract Number: PB0673
Meeting: ISTH 2021 Congress
Background: According to WFH, patients with Hemophilia A should receive factor VIII prophylaxis, as we know that patients with severe disease their unreplacement bleeding rate leads to early joint damage.
The development of inhibitors in patients with haemophilia A occurs around 30% within the first 50 exposures to FVIII concentrate with fatal consequences, there were few therapeutic options for patients with hemophilia and inhibitors who did not undergo ITI; the advent of bypassing agents such as FVIIa and CCPa, on-demand management, improved the bleeding rate.
Emicizumab, a monoclonal antibody that mimics the function of factor VIII has been one of the real and most effective alternatives of recent years in the management of patients with Hemophilia A and inhibitors.
In 2017 Oldenburg et al. reported their results with the use of emicizumab with a 79% decrease in annual bleeding rate (ABR) vs patients using the bypassing agent.
Aims: Described the ABR reduce with emicizumab
Methods: Review ABR pre/post emicizumab
Results: Seven patients with severe hemophilia A and high response titer inhibitors were treated with emicizumab, impregnation dose for 4 weeks and maintenance every 2 weeks, 71% presented life-threatening bleeding before emicizumab. After the monoclonal, 93% decrease joint ABR was observed. During the impregnation period, 28.6% (2/7) presented joint hemorrhage that resolved with 1 dose of FVIIa. All patients have continued with successful rehabilitation.
A patient presented a CNS hemorrhage in the last impregnation week, which required VIIa for 10 days discharged without complications.
Conclusions: Emicizumab significantly reduced the joint annual rate bleeding.Currently assume, bleeding events occurred in the early stages of emicizumab treatment.The use of emicizumab is effective in the hemophilia A with inhibitors management of joint bleeds and has allowing an effective rehabilitation.
Full protection for major bleeding such as CNS is not complete clear, and more studies and patients are required.
To cite this abstract in AMA style:Nunez Toscano M, Lopez-Santiago N, Gonzalez Pedroza MdL, Paredes Aguilera R, Tavera Rodriguez G, Monsivais Orozco A, Maldonado Silva K, Bravo Lindoro A. Decreased Annual Bleeding Rate in Paediatric Patients with Hemophilia a and Inhibitors Treated with Emicizumab vs Bypassing Agents Experience at the National Institute of Pediatrics, Mexico City, Mexico [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/decreased-annual-bleeding-rate-in-paediatric-patients-with-hemophilia-a-and-inhibitors-treated-with-emicizumab-vs-bypassing-agents-experience-at-the-national-institute-of-pediatrics-mexico-city-mexi/. Accessed September 29, 2023.
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