PB0986 - Emicizumab in Acquired Hemophilia A: A Review of Clinical Applications and Outcomes

Background: Acquired Hemophilia A (AHA) is a rare autoimmune disorder characterized by the development of autoantibodies against factor VIII, leading to serious bleeding complications. Traditional management strategies, which involve bypassing agents and immunosuppression, often present challenges in efficacy and safety. Emicizumab, initially developed for congenital hemophilia A, has emerged as a novel therapeutic agent due to its unique mechanism that mimics the function of factor VIII. Its off-label use in AHA has gained attention, prompting a reevaluation of treatment protocols for this patient population.

Aims: This review aims to assess the efficacy and safety of emicizumab in Acquired Hemophilia A by analyzing 141 cases, thereby highlighting its role in treatment and identifying potential risks.

Methods: Adhering to PRISMA guidelines, this systematic review analyzed literature from major databases up to December 2023. Key terms included 'acquired hemophilia A' and 'emicizumab,' focusing on English language articles about AHA patients treated with emicizumab. The review encompassed case reports, open-label clinical trials (phase 2 and phase 3), and retrospective analyses from various medical journals, evaluating each study's methodology, patient demographics, treatment protocols, and outcome measures.

Results: The findings demonstrate emicizumab's efficacy in various treatment contexts, ranging from prophylactic use to the management of AHA. Notable improvements in bleeding control and patient outcomes were observed across studies. However, the review also discusses potential risks and emphasizes the need for individualized treatment approaches.

Conclusion(s): This review recommends caution in using emicizumab for AHA, especially in high-risk groups such as the elderly and cancer patients, due to thrombosis risks. Monitoring endogenous FVIII levels is crucial. Despite the limited data and the need for vigilant patient monitoring, emicizumab shows promise as a therapy for AHA