Abstract Number: PB0683
Meeting: ISTH 2021 Congress
Background: The development of inhibitor in people with HA (PwHA) can increase the annual bleeding rate (ABR), and hemostasis with bypassing agents is not as predictable as with factor VIII (FVIII). Emicizumab is an alternative for such cases.
Aims: We report the first Brazilian woman on emicizumab treatment.
Methods: Medical files were reviewed.
Results: Due to an increase in the partial thromboplastin time at 9 years, she was referred from another center, where she had been treated for Melnick-Needles syndrome. Her FVIII activity was 10.0%. On-demand intravenous FVIII was started (peripheral venous access was difficult to provide due to her body structure). She developed a high-response inhibitor two years later, but immune tolerance induction was not tried because central venous access could not be performed. She was kept on self-infused intravenous activated recombinant factor VII (rFVIIa) episodic treatment.
Between Jan/28/2019 and Jan/28/2020, her ABR was 11.0. Her Functional Independence Score in Hemophilia (FISH) was 21. Total rFVIIa consumption was 375mg. In Jan/28/2020, at 28 years, she received subcutaneous emicizumab attack of 3.0mg/kg once weekly for 4 weeks, and 1.5mg/kg weekly as maintenance. She self-infused emicizumab at home. From Jan/28/2020 to Jan/28/2021, ABR was 0.0 and her FISH increased to 30, although she did not receive emicizumab during 5 consecutive weeks on May-Jun/2020 and 2 consecutive weeks on Jul/2020. Puncture site or thrombotic events were not reported. Her estimated annual consumption of emicizumab was 2,520mg (attack+maintenance during the first year), or 2,340mg, for each additional year without attack doses. The estimated annual cost of treatment increased from US$180,903.22 (US$482.41/mg rFVIIa, purchased by the Brazilian Ministry of Health in 2018) to US$206,551.80-222,440.40 (US$88.27/mg emicizumab, weighted mean of the first 6 purchases from the state Secretary of Health).
Conclusions: Prophylaxis with emicizumab was effective and safe, although treatment costs increased by 14%-23%.
To cite this abstract in AMA style:Camelo RM, Casaretto ES, Castro Tenório MD, Dantas-Silva N, de Figueiredo SS, Álvares-Teodoro J. Twelve Months of Emicizumab Prophylaxis in a Woman with Mild Hemophilia A and Inhibitor: Effectiveness, Safety and Economic Outcome [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/twelve-months-of-emicizumab-prophylaxis-in-a-woman-with-mild-hemophilia-a-and-inhibitor-effectiveness-safety-and-economic-outcome/. Accessed November 29, 2023.
« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/twelve-months-of-emicizumab-prophylaxis-in-a-woman-with-mild-hemophilia-a-and-inhibitor-effectiveness-safety-and-economic-outcome/