Abstract Number: PB0537
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Prophylaxis to prevent spontaneous bleeding and joint disease is the standard of care for people with severe Haemophilia A (PWSHA) in Ireland. In October 2019, Emicizumab became available as an option in Ireland for prophylaxis for PWSHA without inhibitors.
Aims: To offer Emicizumab for prophylaxis in PWSHA without inhibitors, having regard for clinical considerations, safety, personal choice and treatment outcomes.
Methods: PWSHA (n=91) were informed by letter of the availability of Emicizimab as a prophylaxis option. Dedicated clinics were set up to enable adequate time for in-depth clinical review and collaborative discussion about the potential benefits or risks in switching. A standardised process was developed to support patients and the multi-disciplinary team and to ensure a seamless and safe transition to Emicizumab for those who chose to change prophylaxis regimen.
Results: To date, 35 patients have been reviewed at the dedicated clinic and 25 have switched to Emicizumab prophylaxis. Of the remaining 10 patients, seven chose not to switch following consultation, two were non-compliant with recording of home treatment (an exclusion for switching) and one is due to start Emicizumab shortly.
Of the patients who switched to Emicizumab, 24/25 have severe Haemophilia A, one patient has moderate Haemophilia A with a severe bleeding phenotype. Fortnightly maintenance doses are the most common treatment frequency (21/25 patients) with the remaining four patients on weekly maintenance doses.
Since switchover, seven breakthrough bleeds were reported, with five requiring factor replacement (three trauma and two spontaneous bleeds).
Conclusions: A standardised clinical review and consultation process enabled shared decision making with PWSHA regarding their choice of prophylaxis, in line with personal health care goals. 20% of those reviewed chose not to switch to Emicizumab, for clinical reasons or personal choice. Patients receiving Emicizumab prophylaxis demonstrate low bleed rates and improved treatment frequency.
To cite this abstract in AMA style:
Larkin N, Singleton E, Benson J, Boland N, Cleary J, Brady S, Gorman D, Flynn N, Ryan F, Mc Gowen M, Roche S, Bergin C, Mary B, Ryan K, Lavin M, S O Donnell J, O Connell N. Real-world Experience in Introducing Emicizumab Prophylaxis for Adults with Haemophilia A without Inhibitors [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/real-world-experience-in-introducing-emicizumab-prophylaxis-for-adults-with-haemophilia-a-without-inhibitors/. Accessed May 20, 2022.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-world-experience-in-introducing-emicizumab-prophylaxis-for-adults-with-haemophilia-a-without-inhibitors/