Factor VIII associated with lysophosphatidylserine (Lyso-PS) nanoparticles significantly reduces the development of inhibitors in patients with hemophilia A, according to a new study published in the Journal of Thrombosis and Haemostasis. Lyso-PS nanoparticles also improve the survival of factor VIII in plasma, which could increase its clinical utility.

According to the study, a reduction in inhibitor development can be achieved even when the Lyso-PS nanoparticles are administered orally, a much more user-friendly route compared to traditional intravenous administration. 

The development of inhibitors, or antibodies against factor VIII, is one of the main concerns in the clinical management of hemophilia A.

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In order to evaluate the efficacy of factor VIII treatment that is well tolerated by the immune system, a team of researchers led by Sathy V. Balu-Iyer, PhD, developed a factor VIII that was associated with nanoparticles containing Lyso-PS. They tested the efficacy of the new factor VIII by administering it intravenously in a mouse model of hemophilia A. The researchers then administered free factor VIII to the mice weekly. 

The results showed that Lyso-PS-associated factor VIII significantly reduced the development of inhibitors and improved the plasma survival of factor VIII after intravenous administration.

The findings “open avenues for an attractive and promising delivery platform to therapeutically deliver [factor VIII] concentrates while simultaneously preventing the risk of inhibitor development,” Balu-Iyer and the coauthors of the study wrote.

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They added that they are planning further studies to evaluate the potential efficacy of Lyso-PS-associated factor VIII in preventing or stopping bleeding when administered systemically in mouse models of hemophilia A.

Approximately one-third of patients with hemophilia A who are treated with factor VIII develop inhibitors. This new approach could prevent immunogenicity against factor VIII and address clinical demand.


Nguyen NH, Dingman RK, Balu-Iyer SV. Tolerogenic form of factor VIII to prevent inhibitor development in the treatment of hemophilia A. J Thromb Haemost. Published online August 14, 2021. doi:10.1111/jth.15497