Treatment of Severe and Moderately Severe Hemophilia A Without Factor VIII Inhibitors
This protocol covers the management of severe and moderately severe hemophilia A — characterised by factor VIII (FVIII) activity levels below 2 IU/dL — in patients who have no circulating inhibitors to factor VIII.
Clinical Scenario
The target population is defined by FVIII activity <2 IU/dL combined with the absence of factor VIII inhibitors. This combination — significant factor deficiency without inhibitor complication — sets the foundation for the recommended prophylactic strategy outlined in this protocol.
Treatment Approach (Partial Summary)
ISTH guidelines give a strong recommendation for prophylaxis over episodic on-demand treatment in this population. The protocol covers two main prophylaxis strategies for consideration: subcutaneous emicizumab and factor VIII concentrate replacement. The full protocol details the selection criteria, scheduling considerations, and the role of each option — none of which is reproduced here.
References
DOI: 10.1016/j.jtha.2024.05.026
- In individuals with severe and moderately severe hemophilia A without inhibitors, the ISTH Hemophilia Guideline Panel recommends prophylaxis over episodic treatment of bleeding events (strong recommendation, based on moderate-certainty evidence ⨁⨁⨁◯).
- Therefore, we defined hemophilia as severe and moderately severe when individuals enrolled in the study population had FVIII or FIX activity levels <2 IU/dL.
- In individuals with severe and moderately severe hemophilia A without inhibitors, the ISTH Hemophilia Guideline Panel suggests either prophylaxis with emicizumab or prophylaxis with FVIII concentrates (conditional recommendation, based on very low–certainty evidence ⨁◯◯◯).
- Emicizumab may offer a lower treatment burden for patients given its weekly, biweekly, or every 4-week schedule and subcutaneous administration.
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