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
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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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container_title Blood advances
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creator 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
description 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
doi_str_mv 10.1182/bloodadvances.2020001987
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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 &gt;10%. In contrast, 11 of 34 patients with ADAMTS13 activities &lt;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. •ADAMTS13 activities may serve as biomarkers to guide caplacizumab treatment modalities and overall treatment duration.•von Willebrand factor activities may be used for therapeutic drug monitoring of caplacizumab. 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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 &gt;10%. In contrast, 11 of 34 patients with ADAMTS13 activities &lt;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. •ADAMTS13 activities may serve as biomarkers to guide caplacizumab treatment modalities and overall treatment duration.•von Willebrand factor activities may be used for therapeutic drug monitoring of caplacizumab. 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Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Blood advances</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Völker, Linus A.</au><au>Kaufeld, Jessica</au><au>Miesbach, Wolfgang</au><au>Brähler, Sebastian</au><au>Reinhardt, Martin</au><au>Kühne, Lucas</au><au>Mühlfeld, Anja</au><au>Schreiber, Adrian</au><au>Gaedeke, Jens</au><au>Tölle, Markus</au><au>Jabs, Wolfram J.</au><au>Özcan, Fedai</au><au>Markau, Silke</au><au>Girndt, Matthias</au><au>Bauer, Frederic</au><au>Westhoff, Timm H.</au><au>Felten, Helmut</au><au>Hausberg, Martin</au><au>Brand, Marcus</au><au>Gerth, Jens</au><au>Bieringer, Markus</au><au>Bommer, Martin</au><au>Zschiedrich, Stefan</au><au>Schneider, Johanna</au><au>Elitok, Saban</au><au>Gawlik, Alexander</au><au>Gäckler, Anja</au><au>Kribben, Andreas</au><au>Schwenger, Vedat</au><au>Schoenermarck, Ulf</au><au>Roeder, Maximilian</au><au>Radermacher, Jörg</au><au>Bramstedt, Jörn</au><au>Morgner, Anke</au><au>Herbst, Regina</au><au>Harth, Ana</au><au>Potthoff, Sebastian A.</au><au>von Auer, Charis</au><au>Wendt, Ralph</au><au>Christ, Hildegard</au><au>Brinkkoetter, Paul T.</au><au>Menne, Jan</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP</atitle><jtitle>Blood advances</jtitle><addtitle>Blood Adv</addtitle><date>2020-07-14</date><risdate>2020</risdate><volume>4</volume><issue>13</issue><spage>3093</spage><epage>3101</epage><pages>3093-3101</pages><issn>2473-9529</issn><eissn>2473-9537</eissn><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 &gt;10%. In contrast, 11 of 34 patients with ADAMTS13 activities &lt;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. •ADAMTS13 activities may serve as biomarkers to guide caplacizumab treatment modalities and overall treatment duration.•von Willebrand factor activities may be used for therapeutic drug monitoring of caplacizumab. [Display omitted]</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>32634237</pmid><doi>10.1182/bloodadvances.2020001987</doi><tpages>9</tpages><orcidid>https://orcid.org/0000-0003-3235-2994</orcidid><orcidid>https://orcid.org/0000-0001-9843-2132</orcidid><orcidid>https://orcid.org/0000-0003-2823-0847</orcidid><orcidid>https://orcid.org/0000-0002-7862-0993</orcidid><orcidid>https://orcid.org/0000-0002-4461-6128</orcidid><orcidid>https://orcid.org/0000-0003-0661-6984</orcidid><orcidid>https://orcid.org/0000-0003-1600-6581</orcidid><orcidid>https://orcid.org/0000-0002-4287-2080</orcidid><oa>free_for_read</oa></addata></record>
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identifier ISSN: 2473-9529
ispartof Blood advances, 2020-07, Vol.4 (13), p.3093-3101
issn 2473-9529
2473-9537
language eng
recordid cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7362349
source ScienceDirect; PubMed Central
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
title ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP
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