Voelker, Linus A., Kaufeld, Jessica, Miesbach, Wolfgang, Braehler, Sebastian, Reinhardt, Martin ORCID: 0000-0002-7862-0993, Kuehne, Lucas, Muehlfeld, Anja, Schreiber, Adrian, Gaedeke, Jens, Toelle, Markus, Jabs, Wolfram J., Ozcan, Fedai, Markau, Silke, Girndt, Matthias ORCID: 0000-0003-2823-0847, 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, Gaeckler, Anja, Kribben, Andreas, Schwenger, Vedat, Schoenermarck, Ulf, Roeder, Maximilian, Radermacher, Joerg, Bramstedt, Joern, Morgner, Anke, Herbst, Regina, Harth, Ana, Potthoff, Sebastian A., von Auer, Charis, Wendt, Ralph, Christ, Hildegard ORCID: 0000-0003-3235-2994, Brinkkoetter, Paul T. and Menne, Jan (2020). ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP. Blood Adv., 4 (13). S. 3093 - 3102. WASHINGTON: AMER SOC HEMATOLOGY. ISSN 2473-9537

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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.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Voelker, Linus A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kaufeld, JessicaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Miesbach, WolfgangUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Braehler, SebastianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Reinhardt, MartinUNSPECIFIEDorcid.org/0000-0002-7862-0993UNSPECIFIED
Kuehne, LucasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Muehlfeld, AnjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schreiber, AdrianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gaedeke, JensUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Toelle, MarkusUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jabs, Wolfram J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ozcan, FedaiUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Markau, SilkeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Girndt, MatthiasUNSPECIFIEDorcid.org/0000-0003-2823-0847UNSPECIFIED
Bauer, FredericUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Westhoff, Timm H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Felten, HelmutUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hausberg, MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Brand, MarcusUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gerth, JensUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bieringer, MarkusUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bommer, MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Zschiedrich, StefanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schneider, JohannaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Elitok, SabanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gawlik, AlexanderUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gaeckler, AnjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kribben, AndreasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schwenger, VedatUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schoenermarck, UlfUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Roeder, MaximilianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Radermacher, JoergUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bramstedt, JoernUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Morgner, AnkeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Herbst, ReginaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Harth, AnaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Potthoff, Sebastian A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
von Auer, CharisUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wendt, RalphUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Christ, HildegardUNSPECIFIEDorcid.org/0000-0003-3235-2994UNSPECIFIED
Brinkkoetter, Paul T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Menne, JanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-326424
DOI: 10.1182/bloodadvances.2020001987
Journal or Publication Title: Blood Adv.
Volume: 4
Number: 13
Page Range: S. 3093 - 3102
Date: 2020
Publisher: AMER SOC HEMATOLOGY
Place of Publication: WASHINGTON
ISSN: 2473-9537
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
THROMBOTIC THROMBOCYTOPENIC PURPURA; PLASMA-EXCHANGE; RITUXIMAB; EFFICACY; SAFETYMultiple languages
HematologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/32642

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