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

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 his...

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Detalles Bibliográficos
Autores principales: Gavriilaki, Eleni, Nikolousis, Emmanuel, Koravou, Eudoxia-Evaggelia, Dimou-Besikli, Sotiria, Kartsios, Charalampos, Papakonstantinou, Anna, Mpanti, Anastasia, Pontikoglou, Charalampos, Kalpadaki, Christina, Bitsani, Aikaterini, Tassi, Ilianna, Touloumenidou, Tasoula, Chatziconstantinou, Thomas, Papathanasiou, Maria, Syrigou, Antonia, Ztriva, Eleutheria, Kaiafa, Georgia, Mandala, Evdokia, Mellios, Zois, Karakasis, Dimitrios, Kourakli, Alexandra, Symeonidis, Argiris, Kapsali, Eleni, Papadaki, Helen H., Lalayanni, Chrysavgi, Sakellari, Ioanna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10600458/
https://www.ncbi.nlm.nih.gov/pubmed/37901415
http://dx.doi.org/10.3389/fmed.2023.1226114
Descripción
Sumario: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.