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| Item Type: | Article |
|---|---|
| Title: | ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP |
| Creators Name: | Völker, L.A. and Kaufeld, J. and Miesbach, W. and Brähler, S. and Reinhardt, M. and Kühne, L. and Mühlfeld, A. and Schreiber, A. and Gaedeke, J. and Tölle, M. and Jabs, W.J. and Özcan, F. and Markau, S. and Girndt, M. and Bauer, F. and Westhoff, T.H. and Felten, H. and Hausberg, M. and Brand, M. and Gerth, J. and Bieringer, M. and Bommer, M. and Zschiedrich, S. and Schneider, J. and Elitok, S. and Gawlik, A. and Gäckler, A. and Kribben, A. and Schwenger, V. and Schoenermarck, U. and Roeder, M. and Radermacher, J. and Bramstedt, J. and Morgner, A. and Herbst, R. and Harth, A. and Potthoff, S.A. and von Auer, C. and Wendt, R. and Christ, H. and Brinkkoetter, P.T. and Menne, J. |
| Abstract: | 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 <10% at the time of stopping caplacizumab treatment developed a nonfavorable outcome (disease exacerbation or relapse). In some cases, prolongation of the treatment interval to every other day was feasible and resulted in a sustained reduction of von Willebrand factor activity. ADAMTS13 activity measurements are central for a rapid diagnosis in the acute setting but also to tailor disease management. An ADAMTS13 activity-guided approach seems safe for identifying the individual time point when to stop caplacizumab to prevent overtreatment and undertreatment; this approach will result in significant cost savings without jeopardizing the well-being of patients. In addition, von Willebrand factor activity may serve as a biomarker for drug monitoring. |
| Keywords: | ADAMTS13 Gene, Caplacizumab, Biological Markers |
| Source: | Blood Advances |
| ISSN: | 2473-9529 |
| Publisher: | American Society of Hematology |
| Volume: | 4 |
| Number: | 13 |
| Page Range: | 3093-3101 |
| Date: | 14 July 2020 |
| Additional Information: | Copyright © 2020 by The American Society of Hematology |
| Official Publication: | https://doi.org/10.1182/bloodadvances.2020001987 |
| PubMed: | View item in PubMed |
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