Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

被引：100

作者：

Voelker, Linus A. ^{[1
,2
,3
,4
]}

Kaufeld, Jessica ^{[5
]}

Miesbach, Wolfgang ^{[6
]}

Braehler, Sebastian ^{[1
,2
,3
,4
]}

Reinhardt, Martin ^{[5
]}

Kuehne, Lucas ^{[1
,2
,3
,4
]}

Muehlfeld, Anja ^{[7
]}

Schreiber, Adrian ^{[8
,9
]}

Gaedeke, Jens ^{[8
,9
]}

Toelle, Markus ^{[10
,11
,12
,13
]}

Jabs, Wolfram J. ^{[14
]}

Oezcan, Fedai ^{[15
]}

Markau, Silke ^{[16
]}

Girndt, Matthias ^{[16
]}

Bauer, Frederic ^{[17
]}

Westhoff, Timm H. ^{[17
]}

Felten, Helmut ^{[18
]}

Hausberg, Martin ^{[18
]}

Brand, Marcus ^{[19
]}

Gerth, Jens ^{[20
]}

Bieringer, Markus ^{[21
]}

Bommer, Martin ^{[22
]}

Zschiedrich, Stefan ^{[23
]}

Schneider, Johanna ^{[23
]}

Elitok, Saban ^{[24
]}

Gawlik, Alexander ^{[24
]}

Gaeckler, Anja ^{[25
]}

Kribben, Andreas ^{[25
]}

Schwenger, Vedat ^{[26
]}

Schoenermarck, Ulf ^{[27
]}

Roeder, Maximilian ^{[28
]}

Radermacher, Joerg ^{[29
]}

Bramstedt, Joern ^{[30
]}

Morgner, Anke ^{[31
]}

Herbst, Regina ^{[31
]}

Harth, Ana ^{[32
]}

Potthoff, Sebastian A. ^{[33
]}

von Auer, Charis ^{[34
]}

Wendt, Ralph ^{[35
]}

Christ, Hildegard ^{[36
]}

Brinkkoetter, Paul T. ^{[1
,2
,3
,4
]}

Menne, Jan ^{[5
]}

机构：

[1] Univ Cologne, Fac Med, Dept Internal Med 2, Cologne, Germany

[2] Univ Cologne, Fac Med, Ctr Mol Med Cologne, Cologne, Germany

[3] Univ Hosp Cologne, Cologne, Germany

[4] Cologne Cluster Excellence Cellular Stress Respon, Cologne, Germany

[5] Med Sch Hannover, Dept Nephrol & Hypertens, Hannover, Germany

[6] Univ Hosp Frankfurt, Dept Hemostaseol, Hemophilia Ctr, Frankfurt, Germany

[7] Univ Aachen, Transplant Ctr Aachen, Dept Med, Renal Div, Aachen, Germany

[8] Charite Univ Med Berlin, Dept Nephrol & Intens Care Med, Berlin, Germany

[9] Charite, Max Delbruck Ctr Mol Med Helmholtz Assoc, Expt & Clin Res Ctr, Berlin, Germany

[10] Charite Univ Med Berlin, Berlin, Germany

[11] Free Univ Berlin, Berlin, Germany

[12] Humboldt Univ, Berlin, Germany

[13] Berlin Inst Hlth, Dept Nephrol & Intens Care Med, Berlin, Germany

[14] Vivantes Klinikum Friedrichshain, Dept Nephrol, Berlin, Germany

[15] Klinikum Dortmund, Dept Nephrol & Emergency Med, Dortmund, Germany

[16] Martin Luther Univ Halle Wittenberg, Dept Internal Med 2, Halle, Germany

[17] Ruhr Univ Bochum, Marien Hosp Herne, Med Dept 1, Univ Hosp, Herne, Germany

[18] Karlsruhe Gen Hosp, Dept Gen Internal Med Nephrol Rheumatol & Pneumol, Karlsruhe, Germany

[19] Univ Munster, Dept Internal Med D, Munster, Germany

[20] Heinrich Braun Klinikum Zwickau, Dept Internal Med 2, Zwickau, Germany

[21] HELIOS Klin, Dept Cardiol & Nephrol, Berlin, Germany

[22] Alb Fils Kliniken, Dept Internal Med Hematol Oncol Palliat Care & In, Goppingen, Germany

[23] Klinikum Ernst von Bergmann, Dept Nephrol & Endocrinol Diabetol Potsdam, Potsdam, Germany

[24] Klinikum Ernst Bergmann, Dept Nephrol & Endocrinol Diabetol Potsdam, Potsdam, Germany

[25] Univ Essen Duisburg, Univ Hosp Essen, Dept Nephrol, Essen, Germany

[26] Klinikum Stuttgart, Dept Nephrol Hypertens & Autoimmune Disorders, Stuttgart, Germany

[27] Klinikum Univ Munchen, Med Klin & Polikln 4, Nephrol Zentrum, Munich, Germany

[28] Klinikum Landshut, Sect Nephrol, Med Clin 1, Landshut, Germany

[29] Univ Hosp Ruhr Univ Bochum, Ctr Internal Med Nephrol, Johannes Wesling Klinikum Minden, Herne, Germany

[30] Clin Cardiol Nephrol & Intens Care, Bremerhaven, Germany

[31] Klinikum Chemnitz GmbH, Dept Internal Med 3, Chemnitz, Germany

[32] Univ Witten Herdecke, Med Ctr Cologne Merhe, Dept Nephrol Transplantat & Med Intens Care, Cologne, Germany

[33] Heinrich Heine Univ, Med Fac, Dept Nephrol, Dusseldorf, Germany

[34] Johannes Gutenberg Univ Mainz, Dept Hematol Oncol & Pneumol, Univ Med Ctr, Mainz, Rheinland Pfalz, Germany

[35] Hosp St Georg, Dept Nephrol & Kuratorium Dialysis & Transplantat, Leipzig, Germany

[36] Univ Hosp Cologne, Inst Med Stat & Computat Biol, Cologne, Germany

来源：

BLOOD ADVANCES | 2020年 / 4卷 / 13期

关键词：

PLASMA-EXCHANGE; THERAPY;

D O I：

10.1182/bloodadvances.2020001973

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：3085 / 3092

页数：8

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