Abstract Number: PB0964
Meeting: ISTH 2020 Congress
Background: Emicizumab received FDA approval for prophylaxis in hemophilia A patients with inhibitors in November 2017 and for those without inhibitors in October 2018. Hemostatic efficacy of emicizumab in real-world settings, is an area of ongoing interest and clinical relevance.
Aims: Describe a single Center’s experience in adult and pediatric patient annualized bleeding rates (ABR) following transition from factor replacement or bypass therapy to emicizumab prophylaxis.
Methods: Retrospective record review of patients at the Gulf States Hemophilia and Thrombophilia Center of Houston, Texas on factor replacement or bypass therapy who initiated emicizumab following its licensure.
Results: Chart review included 69 patients (29 adults, 40 pediatric) with hemophilia A. Of the 29 adults, 12 (41.4 %) experienced at least one breakthrough bleed event, 5 (27.8 %) of which were trauma induced and 13 (72.2 %) were considered spontaneous. Of the 18 reported breakthrough bleed events 8 (44.4 %) were joint bleeds. An average ABR reduction of 85.4 % (prior average ABR 9.65, post average ABR 1.41) was noted. Of the 40 pediatric patients, 10 (25.0 %) experienced at least one breakthrough bleed event, 4 (20.0 %) of which were trauma induced and 16 (80.0 %) were considered spontaneous. Of the reported 20 breakthrough bleed events 12 (60.0 %) were joint bleeds. An average ABR reduction of 81.8 % (prior average ABR 3.13, post average ABR 0.57) was noted.
Conclusions: The adult population experienced a higher percentage of bleed events on emicizumab prophylaxis (41.4%) as compared to the pediatric population. However, the number of trauma induced versus spontaneous breakthrough bleed events is comparable between the adult and pediatric population. Despite the presence of breakthrough bleed events on emicizumab, average ABR for both the adult and pediatric population was reduced by >80%. Additional observation period with this medication is merited.
To cite this abstract in AMA style:Montanez N, Oldfield D, Gustafson S, Larson J, Rodriguez N, Menon N, Brown D, Escobar M. Use of Emicizumab Prophylaxis in Adult and Pediatric Hemophilia A Patients with and without Inhibitors: Single Center Experience [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/use-of-emicizumab-prophylaxis-in-adult-and-pediatric-hemophilia-a-patients-with-and-without-inhibitors-single-center-experience/. Accessed September 29, 2023.
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