Kuehne, Lucas, Kaufeld, Jessica, Voelker, Linus A., Wendt, Ralph, Schoenermarck, Ulf, Haegele, Holger, Osterholt, Thomas ORCID: 0000-0001-5047-0252, Eichenauer, Dennis A., Bieringer, Markus, Bergwelt-Baildon, Anke, Fischereder, Michael, Buxhofer-Ausch, Veronika, Menne, Jan, Brinkkoetter, Paul T. and Knoebl, Paul ORCID: 0000-0002-7909-7225 (2022). Alternate-day dosing of caplacizumab for immune-mediated thrombotic thrombocytopenic purpura. J. Thromb. Haemost., 20 (4). S. 951 - 961. HOBOKEN: WILEY. ISSN 1538-7836

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Abstract

Background The anti-von Willebrand factor (VWF) nanobody caplacizumab directly prevents the fatal microthrombi formation in immune-mediated thrombotic thrombocytopenic purpura (iTTP), thereby adding a new therapeutic principle to the treatment of this disorder. However, real-world treatment modalities beyond clinical trials remain heterogeneous. Methods Here, we describe the risks and benefits of an alternate-day dosing regimen for caplacizumab by thoroughly analyzing the timing and outcome of this approach in a retrospective cohort of 25 iTTP patients treated with caplacizumab at seven different medical centers in Austria and Germany between 2018 and 2021. Results Alternate-day dosing of caplacizumab appeared feasible and led to persisting normal platelet counts in most patients. Five patients experienced iTTP exacerbations or relapses that led to the resumption of daily caplacizumab application. VWF activity was repeatedly measured in 16 of 25 patients and documented sufficient suppression by caplacizumab after 24 and 48 h in line with published pharmacodynamics. Conclusion Extension of caplacizumab application intervals from daily to alternate-day dosing may be safely considered in selected patients after 3 to 4 weeks of daily treatment. Earlier modifications may be discussed in low-risk patients but require close monitoring for clinical and laboratory features of thrombotic microangiopathy.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Kuehne, LucasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kaufeld, JessicaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Voelker, Linus A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wendt, RalphUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schoenermarck, UlfUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Haegele, HolgerUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Osterholt, ThomasUNSPECIFIEDorcid.org/0000-0001-5047-0252UNSPECIFIED
Eichenauer, Dennis A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bieringer, MarkusUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bergwelt-Baildon, AnkeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fischereder, MichaelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Buxhofer-Ausch, VeronikaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Menne, JanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Brinkkoetter, Paul T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Knoebl, PaulUNSPECIFIEDorcid.org/0000-0002-7909-7225UNSPECIFIED
URN: urn:nbn:de:hbz:38-688426
DOI: 10.1111/jth.15637
Journal or Publication Title: J. Thromb. Haemost.
Volume: 20
Number: 4
Page Range: S. 951 - 961
Date: 2022
Publisher: WILEY
Place of Publication: HOBOKEN
ISSN: 1538-7836
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
CONSENSUSMultiple languages
Hematology; Peripheral Vascular DiseaseMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/68842

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