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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB0665 - 5 Years of Prophylaxis with emicizumab in children and adults with hemophilia A with and without inhibitors in a Portuguese Hemophilia Center – outcomes in bleeding and surgery

    Monday, June 26, 2023
    18:30 – 19:30 ET
    Room: Exhibition
    Background: Prophylaxis with emicizumab, promotes effective hemostasis in persons with hemophilia A (PwHA).

    Aims: To evaluate the efficacy and safety of emicizumab in PwHA followed at a Portuguese Haemophilia Center in a period of 5 years.

    Methods: PwHA data on emicizumab for ≥ 6 months were retrospectively evaluated for cause, frequency and type of bleeding from 12 months before emicizumab till the end of study period.

    Results: Since January 2018, emicizumab was started in 15 patients, 11 children (4 months-12 years) and 4 adults (29 -76 years); 12 with and 3 without inhibitors. In children, median age at initiation of emicizumab was 3.2 (0,5–10) years, and three patients were younger than one year when they started emicizumab. Regarding children without inhibitors, one was an untreated (PUP) and 2 were minimally treated (MTP). Median follow-up time on emicizumab was 40 months (6-60). In a total cumulative time of 611 months, 9 patients (60 %) had no treated bleeding events and, both the 2 MTP and the PUP did not develop breakthrough bleedings. Twelve bleeding events (in 6 patients) were confirmed and treated. These bleedings were mostly related to trauma or surgery (hemarthrosis; mucocutaneous hemorrhage; muscle hematomas and head trauma). In 5 episodes of head trauma (in 2 children), treatment was decided for bleeding prevention, but none of the patients had confirmed intracranial hemorrhage. Three patients experienced spontaneous hemarthrosis. Although target joints were previously reported in 6 patients, after this follow-up period no target joint was identified. Two major orthopedic surgeries and two minor procedures were performed under replacement therapy without any complications. No adverse events were observed.

    Conclusion(s): Although we report a small cohort and a very heterogeneous population, our results show an unexpectedly low bleeding rate in this high risk group. Invasive procedures, including 2 major orthopedic surgeries, were uneventful.