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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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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
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author 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
author_facet 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
author_sort Gavriilaki, Eleni
collection PubMed
description 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.
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spelling pubmed-106004582023-10-27 Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data 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 Front Med (Lausanne) Medicine 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. Frontiers Media S.A. 2023-10-11 /pmc/articles/PMC10600458/ /pubmed/37901415 http://dx.doi.org/10.3389/fmed.2023.1226114 Text en 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. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Medicine
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
Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title_full Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title_fullStr Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title_full_unstemmed Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title_short Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
title_sort caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
topic Medicine
url 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
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