ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP

Introduction of the nanobody caplacizumab was shown to be effective in the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in the acute setting. The official recommendations include plasma exchange (PEX), immunosuppression, and the use of caplacizumab for a minimum of 30 days after...

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Published in:Blood advances 2020-07, Vol.4 (13), p.3093-3101
Main Authors: Völker, Linus A., Kaufeld, Jessica, Miesbach, Wolfgang, Brähler, Sebastian, Reinhardt, Martin, Kühne, Lucas, Mühlfeld, Anja, Schreiber, Adrian, Gaedeke, Jens, Tölle, Markus, Jabs, Wolfram J., Özcan, Fedai, Markau, Silke, Girndt, Matthias, Bauer, Frederic, Westhoff, Timm H., Felten, Helmut, Hausberg, Martin, Brand, Marcus, Gerth, Jens, Bieringer, Markus, Bommer, Martin, Zschiedrich, Stefan, Schneider, Johanna, Elitok, Saban, Gawlik, Alexander, Gäckler, Anja, Kribben, Andreas, Schwenger, Vedat, Schoenermarck, Ulf, Roeder, Maximilian, Radermacher, Jörg, Bramstedt, Jörn, Morgner, Anke, Herbst, Regina, Harth, Ana, Potthoff, Sebastian A., von Auer, Charis, Wendt, Ralph, Christ, Hildegard, Brinkkoetter, Paul T., Menne, Jan
Format: Article
Language:English
Subjects:
ADAMTS13 Protein
Clinical Trials and Observations
Fibrinolytic Agents - therapeutic use
Humans
Purpura, Thrombotic Thrombocytopenic - drug therapy
Retrospective Studies
Single-Domain Antibodies
von Willebrand Factor
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Summary:Introduction of the nanobody caplacizumab was shown to be effective in the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in the acute setting. The official recommendations include plasma exchange (PEX), immunosuppression, and the use of caplacizumab for a minimum of 30 days after stopping daily PEX. This study was a retrospective, observational analysis of the use of caplacizumab in 60 patients from 29 medical centers in Germany. Immunosuppressive treatment led to a rapid normalization of ADAMTS13 activities (calculated median, 21 days). In 35 of 60 patients, ADAMTS13 activities started to normalize before day 30 after PEX; in 11 of 60 patients, the treatment was extended beyond day 30; and in 5 patients, it was extended even beyond day 58 due to persistent autoimmune activity. In 34 of 60 instances, caplacizumab was stopped before day 30 with a favorable outcome whenever ADAMTS13 activities were >10%. In contrast, 11 of 34 patients with ADAMTS13 activities
ISSN:2473-9529
2473-9537
DOI:10.1182/bloodadvances.2020001987