First results of the Phase II TITAN trial: anti-von Willebrand ... - Ablynx

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Major complications of PE • Experience of the Oklahoma TTP-HUS registry, 1996-2008 % of patients 14 12 10 8 6 4 2 0 3 Death Non fatal cardiac arrest 1 2 Catheter insertion complications 12 Systemic infection 7 Catheter obstruction 3 Hypotension Venous thrombosis 2 George et al, Blood 2010 8
Immunosuppressive agents • Corticosteroids – the use of glucocorticoids is based only on clinical experience and case series (Vesely et al Blood 2003, Perotti et al Haematologica 1996, Coppo et al BJH 2006, Cataland et al BJH 2007) – in combination with PE increased complete remission rate and decreased exacerbation and relapse rates (Vesely et al Blood 2003, Coppo et al BJH 2006, Cataland et al BJH 2007, Baduini et al Ann Hematol 2010) • Rituximab – 95% had a complete clinical and laboratory response within 1-3 weeks – mild acute reactions to rituximab infusions easily managed – more serious complications were uncommon – number of PE to remission, inpatient stay and relapse rate reduced (Gutterman LA et al Blood Cells Mol Dis 2002, Galbusera M et al Blood 2005, Herbei L & Venugopal P Clin Adv Hematol Oncol 2006, Scully M et al BJH 2007, Heidel F et al TH 2007, Patino W & Sarode R J Clin Apher 2007, Scully M et al BJH 2008, Bresin E et al TH 2009, Scully M et al Blood 2011) 9
Page 1 and 2: First results of the Phase II TITAN
Page 3 and 4: Clinical symptoms at the first thre
Page 5 and 6: ADAMTS13 • VWF cleaving protease
Page 7: Treatment of acute TTP • Survival
Page 11 and 12: Perspectives • Recombinant ADAMTS
Page 13 and 14: ALX-0081: mode of action in TTP •
Page 15 and 16: ALX-0081 potently neutralises ULVWF
Page 17 and 18: The TITAN trial: study design Inclu
Page 19 and 20: Nanobodies ® - Inspired by nature
Page 21 and 22: Demographic and baseline characteri
Page 23 and 24: PE details • PE treatment - varia
Page 25 and 26: Preliminary observations: RiCo (n =
Page 27 and 28: Preliminary observations: FVIII aci
Page 29 and 30: Preliminary observations: anti-ADAM
Page 31 and 32: Serious adverse events • 2 SAEs r
Page 33: Acknowledgements • Hemophilia and

First results of the Phase II TITAN trial: anti-von Willebrand ... - Ablynx

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