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
BMT Unit - Department of Hematology, G. Papanicolaou Hospital, Thessaloniki, Greece.
Department of Haematology, Athens Medical Center, Athens, Greece.
Front Med (Lausanne). 2023 Oct 11;10:1226114. doi: 10.3389/fmed.2023.1226114. eCollection 2023.
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, = 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 ( < 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.
鉴于卡泊珠单抗的真实世界数据有限，我们开展了一项多中心真实世界研究，旨在与历史对照相比，评估卡泊珠单抗在免疫性血栓性血小板减少性紫癜（iTTP）中的安全性和疗效。我们研究了70例患者：23例接受卡泊珠单抗治疗，47例为历史对照组。除2例拒绝血浆置换的患者外，所有发作期均进行了血浆置换。与历史对照组相比，利妥昔单抗作为一线治疗在卡泊珠单抗组更为常见。卡泊珠单抗（每日10 mg）在初始诊断后第7天（1 - 43天）中位数时间开始给药，共给药32次（6 - 47次）。在卡泊珠单抗组，中位数为12例（8 - 23例）患者需要进行血浆置换，而对照组为14例（6 - 32例）。给予卡泊珠单抗未产生任何3级并发症或重大出血事件。自iTTP诊断后中位数19.0（2.6 - 320）个月，发生了5例死亡（对照组4例，卡泊珠单抗组1例，P = 0.310）。卡泊珠单抗治疗的患者在治疗结束时实现了早期血小板正常化和ADAMTS13活性正常化。仅2/23例（9%）接受卡泊珠单抗治疗的患者出现复发，而历史对照组为29/47例（62%）（P < 0.001）。总体而言，卡泊珠单抗在治疗iTTP方面是安全有效的，包括对血浆置换难治、再次给药以及既往未接受血浆置换治疗的病例。未观察到重大出血事件。以ADAMTS13为指导停止给药确保了低复发率。
