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
Gavriilaki, Eleni
Nikolousis, Emmanuel
Koravou, Eudoxia-Evaggelia
Dimou-Besikli, Sotiria
Kartsios, Charalampos
Papakonstantinou, Anna
Mpanti, Anastasia
Pontikoglou, Charalampos
Kalpadaki, Christina
Bitsani, Aikaterini
Citations
Altmetric:
Affiliation
Other Contributors
Publication date
2023-10-11
Subject
Collections
Research Projects
Organizational Units
Journal Issue
Abstract
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.
Citation
Gavriilaki E, Nikolousis E, Koravou EE, Dimou-Besikli S, Kartsios C, Papakonstantinou A, Mpanti A, Pontikoglou C, Kalpadaki C, Bitsani A, Tassi I, Touloumenidou T, Chatziconstantinou T, Papathanasiou M, Syrigou A, Ztriva E, Kaiafa G, Mandala E, Mellios Z, Karakasis D, Kourakli A, Symeonidis A, Kapsali E, Papadaki HH, Lalayanni C, Sakellari I. Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data. Front Med (Lausanne). 2023 Oct 11;10:1226114. doi: 10.3389/fmed.2023.1226114. PMID: 37901415; PMCID: PMC10600458.
Type
Article