N. De Beule MD, PhD, R. Schots MD, PhD, A. De Becker MD
SUMMARY
Acquired or immune-mediated thrombotic thrombocytopenic purpura (aTTP) is a life-threatening auto-immune disorder caused by a functional deficiency of the von Willebrand factor-cleaving protease ADAMTS13, leading to thrombotic microangiopathy. The introduction of plasma-exchange has reduced mortality from over 90% to 10–20%. However, over the last two decades the treatment outcomes have not changed substantially. Caplacizumab, a humanised nanobody directed to the A1 domain of VWF, inhibits this lethal thrombotic cascade and is therefore essential for symptom control and prevention of irreversible end-organ damage. Both TITAN and HERCULES trials demonstrated that treatment with caplacizumab significantly reduced mean duration of hospitalisation and number of days of plasma-exchange. Moreover, no deaths were observed in caplacizumab-treated patients. Therefore, we can state that caplacizumab has changed the treatment paradigm of aTTP.
BJH - volume 6, issue Abstract Book BHS, january 2015
K. De Veirman , J. van Ginderachter , N. De Beule MD, PhD, S. Lub , A. Maes , E. De Bruyne , E. Menu , I. Van Riet PhD, K. Vanderkerken PhD, E. Van Valckenborgh PhD