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., Oezcan, 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). Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood Adv., 4 (13). S. 3085 - 3093. WASHINGTON: AMER SOC HEMATOLOGY. ISSN 2473-9537

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Abstract

Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare but life-threatening condition. In 2018, the nanobody caplacizumab was approved for the treatment of adults experiencing an acute episode of aTTP, in conjunction with plasma exchange (PEX) and immunosuppression for a minimum of 30 days after stopping daily PEX. We performed a retrospective, observational analysis on the use of caplacizumab in 60 patients from 29 medical centers in Germany during acute disease management. Caplacizumab led to a rapid normalization of the platelet count (median, 3 days; mean 3.78 days). One patient died after late treatment initiation due to aTTP-associated complications. In 2 patients with initial disease presentation and in 4 additional patients with laboratory signs of an exacerbation or relapse after the initial therapy, PEX-free treatment regimens could be established with overall favorable outcome. Caplacizumab is efficacious in the treatment of aTTP independent of timing and ancillary treatment modalities. Based on this real-world experience and published literature, we propose to administer caplacizumab immediately to all patients with an acute episode of aTTP. Treatment decisions regarding the use of PEX should be based on the severity of the clinical presentation and known risk factors. PEX might be dispensable in some patients.

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
Oezcan, 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:38-326412
DOI: 10.1182/bloodadvances.2020001973
Journal or Publication Title: Blood Adv.
Volume: 4
Number: 13
Page Range: S. 3085 - 3093
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
PLASMA-EXCHANGE; THERAPYMultiple languages
HematologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/32641

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