Abstract Number: PB1086
Meeting: ISTH 2020 Congress
Background: Neutralizing inhibitors against factor VIII has been a challenge in hemophilia due to inappropriate control of bleedings with bypass agents, development of arthropathy and need of central venous access.
An emergent monoclonal antibody “emicizumab” has arrived as a technology that provides prothrombinase activity, binding Factor IXa and Factor X with bispecific domains and solving unmet medical needs like subcutaneous administration and long acting efficacy.
The only intervention identified for inhibitor eradication has been immunotolerance and even in the times of emicizumab remains as an important approach to be considered in real life medical practice.
Aims: To describe patients with Hemophilia A under 18 years old with high inhibitors titers in therapy with emicizumab plus immunotolerance (Adapted Regimen: FIT – Future of Immunotolerance Treatment Group).
Methods: Clinical data of patients were obtained and analyzed from registry of the hemophilia center “IPS Especializada”, informed consent was obtained for every patient and approval of institutional medical ethics committee was provided.
Results: Three male patients with Hemophilia A and high inhibitors titers were included, once patients tracing started, their age were 1, 4 and 15 years old, theirs inhibitor levels were 35, 16 and 100 UBethesda respectively, all of them used bypass agents as prophylaxis and two of them were receiving immunotolerance induction, additional population characteristics Table 1. Every patient was proposed to change therapy to emicizumab plus immunotolerance with Factor VIII (50 Ui/kg thrice a week), two of them accepted the new scheme without develop new bleedings, one of them reached negative inhibitors five months later and the other one has remained with high inhibitor titer. No adverse events have been reported.
Conclusions: New scheme of therapy with emicizumab and immunotolerance is feasible in children with Hemophilia A plus inhibitors, achieving the improvement of bleeding phenotype and committed with inhibitors eradication.
To cite this abstract in AMA style:Ardila J, Argüello J, Martinez G, Wilches J, Arias D. Immune Tolerance Induction in Children with Hemophilia A, in Times of Emicizumab: A Real Life Experience [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/immune-tolerance-induction-in-children-with-hemophilia-a-in-times-of-emicizumab-a-real-life-experience/. Accessed March 4, 2024.
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