Abstract Number: PB0539
Meeting: ISTH 2021 Congress
Background: In Portugal, emicizumab, although only approved for persons with hemophilia A with inhibitors (PwHAi), changed the mindset of prophylaxis in haemophilia.
Aims: To evaluate the clinical efficacy of emicizumab prophylaxis in PwHAi followed at our center.
Methods: Data on PwHAi on prophylaxis with emicizumab for the last 3 years, were retrospectively retrieved from the medical records. Other than annual bleeding rates (BR), an evaluation of episodes responsible for visits to the emergency room (ER) and for hospitalization was also done.
Results: Since January 2018, emicizumab was started in 9 patients (8 children, 1 adult) and the median duration of therapy was 29months (18-38). Age at initiation of emicizumab ranged from 4 months to 27 years, median 6.5years. 3 patients had received immune tolerance induction (ITI) without success and were on prophylaxis with bypassing agents (BPA). On emicizumab, no patient experienced spontaneous bleeding and 7 patients had no treated bleeding events. The ABR with emicizumab was 0.3 (0-2) vs 10.4 (1-24) with prior BPA, representing a 97% reduced risk of bleeding. 2 patients were occasionally treated with rFVIIa for trauma (hemarthrosis; epistaxis; traumatic lip bleeding; 2 muscle hematomas and head trauma), and 1 and for prevention of bleeding before 1 minor procedure. In the period between 2016 and 2017, 3/5 of our PWHi had at least one hospitalization and were responsible for 22% of all hospital admissions in PWSevereH. However, since the beginning of emicizumab, there were no hospitalizations. Regarding visits to the ER, before 2018 PWHi accounted for about 10% of these visits, but this also changed after prophylaxis with emicizumab, with only 6 visits in this 3-year period.
Conclusions: In our study, we find that the efficacy of emicizumab is well demonstrated by the low BR, as well as by the almost inexistent visits to the ER and by the lack of hospitalizations.
To cite this abstract in AMA style:Campaniço S, Rodrigues F, Pereira A, Rocha E, Cevadinha G, Catarino C. Is Zero Bleeds in PWH an Achievable Goal? 3 Years of Emicizumab in Persons with Hemophilia A and Inhibitors in a Portuguese Hemophilia Treatment Centers [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/is-zero-bleeds-in-pwh-an-achievable-goal-3-years-of-emicizumab-in-persons-with-hemophilia-a-and-inhibitors-in-a-portuguese-hemophilia-treatment-centers/. Accessed September 24, 2021.
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