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 TITLE=Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data JOURNAL=Frontiers in Medicine VOLUME=10 YEAR=2023 URL=https://www.frontiersin.org/journals/medicine/articles/10.3389/fmed.2023.1226114 DOI=10.3389/fmed.2023.1226114 ISSN=2296-858X 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.