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Possible new approach to treating a life-threatening blood disorder

JCI Journals

Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease of the blood system. The condition is caused by the presence of ultralarge multimers of the protein von Willebrand factor, which promote the formation of blood clots (thrombi) in small blood vessels throughout the body. Current treatments are protracted and associated with complications. However, a team of researchers, led by José López, at the Puget Sound Blood Center, Seattle, has generated data in mice that suggest that the drug N-acetylcysteine (NAC), which is FDA approved as a treatment for chronic obstructive lung disease and as an antidote for toxicity due to acetaminophen (paracetamol), might provide a rapid and effective treatment for patients with TTP through its ability to decrease the size of von Willebrand factor multimers.

In an accompanying commentary, Michael Berndt and Robert Andrews, concur with the conclusions of López and colleagues, although they caution that there are a number of caveats to the view that NAC could be used to treat patents with TTP.


TITLE: N-acetylcysteine reduces the size and activity of von Willebrand factor in human plasma and mice

José A. López
Puget Sound Blood Center, Seattle, Washington, USA.
Phone: 206.398.5930; Fax: 206.587.6056; E-mail:

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TITLE: Thrombotic thrombocytopenic purpura: reducing the risk?

Michael C. Berndt
Dublin City University, and Royal College of Surgeons in Ireland, Dublin, Ireland.
Phone: 353.1.7007658; Fax: 353.1.7006558; E-mail:

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