Refractory Auto-Immune Thrombotic Thrombocytopenic Pupura Successfully Treated With Caplacizumab

Frontiers in Medicine
Chloé MellazaJean-François Augusto

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare thrombotic microangiopathy characterized by mechanical hemolytic anemia, profound thrombocytopenia, and neurological manifestations. Acquired auto-immune TTP, the most prevalent cause of TTP, is induced by the presence of inhibitory anti-ADAMTS13 auto-antibodies. Modern treatment of acquired TTP relies on plasma exchange, rituximab, and steroids. Caplacizumab (Cablivi®), a humanized single-variable domain immunoglobulin that targets the A1 domain of the ultra-large von Willebrand factor, inhibits the interaction between ultra-large vWFand platelets. In two clinical trials, caplacizumab, in addition to conventional treatment, shortened the delay to platelet count normalization in comparison to conventional treatment plus placebo, without increasing significantly hemorrhagic complications. Moreover, caplacizumab was associated with reduced occurrence of a secondary endpoint associating death, TTP recurrence, and major thromboembolic events. Here, we report the off-label use of caplacizumab in a 68-year-old patient with confirmed acquired TTP, severe thrombocytopenia, and generalized tonic-clonic seizures requiring mechanical ventilation and admission in the intensive care unit. ...Continue Reading

References

Aug 24, 2005·British Journal of Haematology·M C Kappers-KlunneA Brand
Mar 13, 2012·Transfusion·François BeloncleUNKNOWN Thrombotic Microangiopathies Reference Center
Mar 19, 2015·Blood·Farzana A Sayani, Charles S Abrams
Feb 11, 2016·The New England Journal of Medicine·Flora PeyvandiUNKNOWN TITAN Investigators
Feb 11, 2016·The New England Journal of Medicine·Agnès Veyradier
Apr 7, 2017·Nature Reviews. Disease Primers·Johanna A Kremer HovingaKaren Vanhoorelbeke
Apr 19, 2017·Blood·Bérangère S JolyAgnès Veyradier
Mar 24, 2018·Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis·Ernesto VignaMassimo Gentile
Jan 10, 2019·The New England Journal of Medicine·Marie ScullyUNKNOWN HERCULES Investigators

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