Treatment of Severe Hemophilia A Without Inhibitors in Resource-Limited Settings
This protocol covers management of patients with severe hemophilia A — factor VIII activity below 1 IU/dL — who have no factor VIII inhibitors, and who are in settings where standard-dose prophylaxis is not accessible.
Clinical scenario: Severe hemophilia A (factor VIII activity <1 IU/dL) without factor VIII inhibitors, in resource-limited settings where standard-dose prophylaxis is not possible.
Recommended Approach
In this setting, current guidance favours a prophylactic strategy using a low-dose factor concentrate over reactive treatment of individual bleeding episodes. The specific regimen — including schedule and dosing parameters — is detailed in the full structured protocol.
References
DOI: 10.1016/j.jtha.2024.05.026
In resource-limited settings in which the use of standard-dose prophylaxis for severe hemophilia A without inhibitors is not possible, the ISTH Hemophilia Guideline Panel suggests prophylaxis with low-dose FVIII concentrates over episodic treatment of bleeding events (conditional recommendation, based on very low–certainty evidence ⨁◯◯◯).
In contrast, low-dose prophylaxis with FVIII was defined as 10 IU/kg, 2 times per week, according to the studies included in recommendation 4, both of which used plasma-derived FVIII.
View source ↗