Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
Eleni Gavriilaki, Emmanuel Nikolousis, Eudoxia‐Evaggelia Koravou, Sotiria Dimou‐Besikli, Charalampos Kartsios, Anna Papakonstantinou, A. Mpanti, Charalampos Pontikoglou, Christina Kalpadaki, Aikaterini Bitsani, Ilianna Tassi, Tasoula Touloumenidou, Thomas Chatziconstantinou, Μαρία Παπαθανασίου, Antonia Syrigou, Eleutheria Ztriva, Georgia Kaiafa, Evdokia Mandala, Zois Mellios, Dimitrios Karakasis, Alexandra Kourakli, Argiris Symeonidis, Eleni Kapsali, Helen Papadaki, Chrysavgi Lalayanni, Ioanna Sakellari
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.