R. Camelo1, N. Dantas-Silva2, J. Alvares-Teodoro3

1Universidade Federal de Minas Gerais, Faculty of Medicine, Belo Horizonte, Brazil; 2Fundação HEMOMINAS, Belo Horizonte, Brazil; 3Universidade Federal de Minas Gerais, Faculty of Pharmacy, Belo Horizonte, Brazil

BACKGROUND

Emicizumab (MC-Ab) is a humanized bispecific antibody which binds to factors IX-activated and X, speeding up the activation of factor X. It has bypassed some unmet needs in hemophilia A (HA) treatment, such as regimen (once weekly up to once monthly infusion) and route of administration (subcutaneous). Although it is an effective non-replacement alternative in the prophylaxis of people with HA (PwHA) with (PwHAi) or without inhibitor, its safety has not been clarified yet, and a few cases of thrombosis and development of anti-MC-Ab antibody have been described.
The aim of this project is to create a national registry to follow up PwHA receiving MC-Ab.

METHODS

EMCase is an observational study and any PwHA receiving MC-Ab can be included. The treatment will be decided among the patient, the physician and the interdisciplinary team of the hemophilia treatment center (HTC) of each Brazilian state (Figure). The research group will develop a brochure with suggestions on classical outcome assessment tools (e.g., bleeding rate, joint health, absenteeism, adherence, quality of life and mortality) which can be evaluated as the judgement of the HTC team. Outcome data, laboratory results and therapeutic progression will be compiled yearly over 10 years. Pharmacovigilance and economic analyses will also be included. Finally, a national guidance will be developed.

RESULTS

In Brazil, MC-Ab was approved in 2018 only for treatment of PwHAi*. In 2015, there were 250 patients on immune tolerance and the failure rate of this treatment has been described about 20%. Consequently, we expect to register at least 50 PwHAi.

*MC-Ab was approved for PwHA after the acceptance of this abstract.

Figure. Brazilian states which are already participating in the study (in red).

CONCLUSION

We expect to establish some outcome assessment tools and laboratory tests to aid the interdisciplinary team to manage hemophilia treatment with MC-Ab as well as to help to clarify the safety of this bispecific antibody.

FINANCIAL SUPPORT
There was not financial support for this research.

DISCLOSURES
There was not conflict of interest for this research.

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