Abstract Number: PB0687
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia
Background: Emicizumab has been approved recently in Brazil for prophylaxis of children with hemophilia A and inhibitors. However, before the incorporation, patients accessed the medicine by judicialization.
Aims: We aimed to describe the 8-month experience of emicizumab prophylaxis in a boy with severe hemophilia A and inhibitor.
Methods: Patient medical files were reviewed.
Results: He was diagnosed at 7 months after a large subcutaneous hematoma developed during venous puncture. Prophylaxis was not started due to poor venous access and frequent subcutaneous/muscular hematomas when venous punctures were performed. Central venous device was not implanted, because the patient did not have prompt access to a Vascular Surgeon. High-response inhibitor (17.5BU/mL) was diagnosed at 1.5 year, after 20 exposure days. Immune tolerance induction or bypassing agent prophylaxis were not indicated due to the difficulty of his venous route. In the following 9 months, his annualized bleeding rate was 17.3, including subcutaneous/muscular hematomas after venous punctures. He has been hospitalized many times to treat muscular bleedings (gluteus, thighs, and calves). After judicialization, emicizumab was initiated by four attacking doses (3.0mg/kg/wk) and maintenance thereafter (1.5mg/kg/wk). Emicizumab was stopped for 8 weeks, because the Health Secretary did not purchase the product. The patient had hemarthrosis in the right knee, which was treated with recombinant activated factor VII. Emicizumab was resumed as the maintenance regimen. No treated bleeding, puncture site event, nor thrombosis have been reported during emicizumab treatment. He did not receive clotting factors to treat many bruises, one jugal mucosa hematoma after biting, and one subgaleal hematoma after falling, which solved spontaneously.
Conclusions: We conclude that emicizumab was safe and effective as prophylaxis, and the boy has been benefitting from maintaining its use. We expect to start immune tolerance induction as soon as he has better vein access.
To cite this abstract in AMA style:
Camelo RM, Palis M, de Cerqueira MAF, Dantas-Silva N, Álvares-Teodoro J. Successful Experience of Emicizumab Prophylaxis in a Child with Severe Hemophilia A and Inhibitor without Previous Immune Tolerance Induction [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/successful-experience-of-emicizumab-prophylaxis-in-a-child-with-severe-hemophilia-a-and-inhibitor-without-previous-immune-tolerance-induction/. Accessed November 29, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/successful-experience-of-emicizumab-prophylaxis-in-a-child-with-severe-hemophilia-a-and-inhibitor-without-previous-immune-tolerance-induction/