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

Autor: Eleni Gavriilaki, Emmanuel Nikolousis, Eudoxia-Evaggelia Koravou, Sotiria Dimou-Besikli, Charalampos Kartsios, Anna Papakonstantinou, Anastasia Mpanti, Charalampos Pontikoglou, Christina Kalpadaki, Aikaterini Bitsani, Ilianna Tassi, Tasoula Touloumenidou, Thomas Chatziconstantinou, Maria Papathanasiou, Antonia Syrigou, Eleutheria Ztriva, Georgia Kaiafa, Evdokia Mandala, Zois Mellios, Dimitrios Karakasis, Alexandra Kourakli, Argiris Symeonidis, Eleni Kapsali, Helen H. Papadaki, Chrysavgi Lalayanni, Ioanna Sakellari
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Frontiers in Medicine, Vol 10 (2023)
Druh dokumentu: article
ISSN: 2296-858X
DOI: 10.3389/fmed.2023.1226114
Popis: 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
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