Abstract Number: PB1029
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: In Portugal, emicizumab, a bispecific monoclonal antibody that mimics FVIII, is only approved for the prevention of bleeding in persons with hemophilia A with FVIII inhibitors (PwHAi).
Aims: To evaluate the clinical efficacy and safety of emicizumab prophylaxis in PwHAi followed at 4 Portuguese Hemophilia Comprehensive Care Centers.
Methods: Emicizumab prophylaxis was evaluated in PwHAi previously receiving episodic/prophylactic bypassing agents (BPAs) treatment. Intraindividual comparison of bleeding rates (BR) and presence of target joints before and after starting emicizumab was done.
Results: Since January 2018, emicizumab prophylaxis was started in 17 patients, 10 children (4 months-12years) and 7 adults (27-55years) who were followed for a median of 71.4 weeks. Immune tolerance induction (ITI) was performed without success in 6 patients, and in the 5 younger children ITI was postponed mainly due to parents’ refusal to CVL insertion. On emicizumab prophylaxis no patient experienced spontaneous bleeding and 12 patients had no treated bleeding events. The BR with emicizumab was 0.3 vs 9.1 with prior BPA, representing a 97% reduced risk of bleeding with emicizumab. Comparison of BR is more difficult in the 5 children in whom emicizumab was started at a very young age, but bleeding events in this group were unexpectedly rare. Although target joints were previously reported in 9 patients, after this follow-up period no target joint was identified in any patient. 7 patients were occasionally treated with recombinant activated FVII for trauma (hemarthrosis, epistaxis, 2 muscle hematomas and head trauma), bleeding after 2 minor surgeries and for prevention of bleeding before 1 minor procedure. No significant adverse events, namely thrombotic events, were observed.
Conclusions: Our experience suggests that emicizumab prophylaxis is effective and safe and leads to significantly less bleeding events and to resolution of target joints. We all are, however, aware how important is to maintain an active vigilance.
To cite this abstract in AMA style:
Catarino C, Rodrigues F, Pereira A, Campaniço S, Santos A, Antunes M, Diniz MJ, Lopes M, Fernandes S, Carvalho M, Salvado R, Rocha E, Cevadinha G. Prophylaxis with Emicizumab in Children and Adults with Hemophilia A and Inhibitors: Real-World Data of 17 Patients from 4 Portuguese Hemophilia Treatment Centers [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/prophylaxis-with-emicizumab-in-children-and-adults-with-hemophilia-a-and-inhibitors-real-world-data-of-17-patients-from-4-portuguese-hemophilia-treatment-centers/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/prophylaxis-with-emicizumab-in-children-and-adults-with-hemophilia-a-and-inhibitors-real-world-data-of-17-patients-from-4-portuguese-hemophilia-treatment-centers/