Abstract Number: PB0637
Meeting: ISTH 2021 Congress
Background: Emicizumab is a biespecific,humanized, monoclonal antibody that restores the function of missing FVIIIa in patientes with hemophilia A (PwHA) .In Mexico, Emicizumab is approved for PwHA with and without inhibitors, but the experience in PwHA with special characteristics is limited.
Aims: The objective of this retrospective study was to describe our real-world experience in prescribing Emicizumab prophylaxis in 10 PwHA with special characteristics treated in private practice in Mexico.
Methods: Eligible patients for our retrospective analysis included patients with severe hemophilia A treated with Emicizumab in ptivate practice in Mexico between September 2019 to December 2020. Data collected included: age, bleeding,and treatment history before and after initiation on Emicizumab, the reason for switching,comorbidities, and adverse events.
Results: We presented the data of ten patients, median age 8 years, all males, who attended our clinics. All of them received Emicizumab for more than 4 months. Nine switched from FVIII to Emicizumab and 8, 9 were on prophylaxis or ITI with either rFVIII or pdFVIII. Patient 10 was a PUP and received FVIII secondary to bleeding and he started on Emicizumab afterward. Patient 1, received ITI for a year without success, he is currently on Emicizumab + ITI and he reported 0 bleeds since then. Patient 4 has obesity and autism and patients 6-8 are siblings, all of their caregivers reported improved adherence with the SQ therapy started. Patient 8 has been treated with cryoprecipitates and he has HIV, HCV, and chronic arthropathy. All patients improve their bleeding rate, 80% have 0 bleeds. No significant adverse events were observed.
Prophylaxis with Emicizumab was efficacious and well-tolerated in our PwHA with special characteristics. All of them improve their bleeding rate and adherence. More data are needed on the use of Emicizumab, particularly in PUPs and patients with comorbidities concerning safety and efficacy.
To cite this abstract in AMA style:Pompa Garza MT, Arias Aranda A, Miranda Madrazo R, Padilla Duron N, Gómez González CS. Real World Experience with Emicizumab with Hemophilia A Treated in Private Practice in Mexico [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/real-world-experience-with-emicizumab-with-hemophilia-a-treated-in-private-practice-in-mexico/. Accessed September 24, 2021.
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