Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data

Frontiers in Medicine

Medicine

Georgia Kaiafa¹  Eleutheria Ztriva¹  Evdokia Mandala²  Eleni Gavriilaki³  Tasoula Touloumenidou³  Maria Papathanasiou³  Chrysavgi Lalayanni³  Thomas Chatziconstantinou³  Ioanna Sakellari³  Antonia Syrigou³  Eudoxia-Evaggelia Koravou³  Sotiria Dimou-Besikli³  Emmanuel Nikolousis⁴  Helen H. Papadaki⁴  Dimitrios Karakasis⁵  Zois Mellios⁵  Anastasia Mpanti⁶  Eleni Kapsali⁷  Ilianna Tassi⁷  Charalampos Pontikoglou⁸  Christina Kalpadaki⁸  Argiris Symeonidis⁹  Alexandra Kourakli⁹  Aikaterini Bitsani^1,10  Charalampos Kartsios^1,11  Anna Papakonstantinou^1,12 

[1] 1st Medical Propaedeutic Department of Internal Medicine, Aristotle University of Thessaloniki, Thessaloniki, Greece;4th Department of Internal Medicine, Aristotle University of Thessaloniki, Thessaloniki, Greece;BMT Unit - Department of Hematology, G. Papanicolaou Hospital, Thessaloniki, Greece;Department of Haematology, Athens Medical Center, Athens, Greece;Department of Hematology, Evangelismos Hospital, Athens, Greece;Department of Hematology, Papageorgiou Hospital, Thessaloniki, Greece;Department of Hematology, University Hospital, Ioannina, Greece;Department of Hematology, University of Crete School of Medicine, Crete, Greece;Division of Hematology, Department of Internal Medicine, University Hospital of Patras, Patras, Greece;First Department of Internal Medicine, LAIKO General Hospital, Athens, Greece;Heartlands Hospital, Birmingham, United Kingdom;Medical School, Aristotle University of Thessaloniki, Thessaloniki, Greece;

关键词: caplacizumab;    thrombotic thrombocytopenic purpura;    plasma exchange;    ADAMTS13;    multicenter real-world study;   

DOI  :  10.3389/fmed.2023.1226114

 received in 2023-05-20, accepted in 2023-09-27,  发布年份 2023

来源: Frontiers

【 摘 要 】

Given the limited real-world data of caplacizumab, our multicenter real-world study was designed to assess the safety and efficacy of caplacizumab in immune thrombotic thrombocytopenic pupura (iTTP), compared to historic controls. We have studied 70 patients: 23 in the caplacizumab and 47 in the historic control group. Plasma exchange was applied in all episodes except for two patients that denied plasma exchange. Rituximab as first-line treatment was more common in the caplacizumab group compared to historic control. Caplacizumab (10 mg daily) was given at a median on day 7 (1–43) from initial diagnosis for 32 (6–47) dosages. In the caplacizumab group, a median of 12 (8–23) patients required plasma exchange sessions versus 14 (6–32) in the control group. Caplacizumab administration did not produce any grade 3 complications or major hemorrhagic events. After a median of 19.0 (2.6–320) months since the iTTP diagnosis, 5 deaths occurred (4 in the control group and 1 in the caplacizumab group, p = 0.310). Caplacizumab patients achieved early platelet normalization and ADAMTS13 activity normalization at the end of treatment. Relapse was observed only in 2/23 (9%) caplacizumab patients, compared to 29/47 (62%) historic controls (p < 0.001). Overall, caplacizumab is safe and effective in treating iTTP, including cases refractory to plasma exchange, re-administration, and cases without previous plasma exchange treatment. No major hemorrhagic events were observed. Cessation of dosing guided by ADAMTS13 has ensured a low relapse rate.

【 授权许可】

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Copyright © 2023 Gavriilaki, Nikolousis, Koravou, Dimou-Besikli, Kartsios, Papakonstantinou, Mpanti, Pontikoglou, Kalpadaki, Bitsani, Tassi, Touloumenidou, Chatziconstantinou, Papathanasiou, Syrigou, Ztriva, Kaiafa, Mandala, Mellios, Karakasis, Kourakli, Symeonidis, Kapsali, Papadaki, Lalayanni and Sakellari.