Mingot-Castellano María-Eva, García-Candel Faustino, Martínez-Nieto Jorge, García-Arroba José, de la Rubia-Comos Javier, Gómez-Seguí Inés, Paciello-Coronel María-Liz, Valcárcel-Ferreiras David, Jiménez Moraima, Cid Joan, Lozano Miquel, García-Gala José-María, Angós-Vazquez Sonia, Vara-Pampliega Miriam, Guerra-Domínguez Luisa, Ávila-Idrobo Laura-Francisca, Oliva-Hernandez Ana, Zalba-Marcos Saioa, Tallón-Ruiz Inmaculada, Ortega-Sánchez Sandra, Goterris-Viciedo Rosa, Moreno-Jiménez Gemma, Domínguez-Acosta Lourdes, Araiz-Ramírez María, Hernández-Mateos Luis, Flores-Ballesteros Elena, Del Río-Garma Julio, Pascual-Izquierdo Cristina
Department of Hematology, Hospital Universitario Virgen del Rocío, Seville, Spain.
Instituto de Biomedicina de Sevilla, Seville, Spain.
Blood. 2024 May 2;143(18):1807-1815. doi: 10.1182/blood.2023022725.
Caplacizumab prevents the interaction between von Willebrand factor and platelets and is used to treat immune thrombotic thrombocytopenic purpura (iTTP). Its administration has been associated with a delay in ADAMTS13 activity restoration after plasma exchange (PEX) suspension. We analyzed the outcomes of 113 iTTP episodes, 75 of which were treated with caplacizumab, in 108 patients from the Spanish Registry of Thrombotic Thrombocytopenic Purpura. Caplacizumab shortened the time to platelet count normalization and reduced PEX requirement, exacerbations, and relapses. There was no difference in the time to achieve ADAMTS13 activity ≥20% after PEX end between caplacizumab-treated and nontreated episodes (median [interquartile range], 14.5 [7.7-27.2] vs 13.0 [8.0-29.0] days, P = .653). However, considering the 36 episodes in which caplacizumab was started ≤3 days after iTTP diagnosis, the time for ADAMTS13 restoration from PEX end was higher than in those episodes in which caplacizumab was started >3 days after iTTP diagnosis (20.0 [12.0-43.0] vs 11.0 [3.5-20.0] days, P = .003) or than in non-caplacizumab-treated episodes (P = .033). This finding could be related to a significantly shorter duration of PEX in early caplacizumab-treated episodes than in late caplacizumab-treated episodes (5.5 [4.0-9.0] vs 15.0 [11.0-21.5] days, P < .001) or non-caplacizumab-treated episodes (11.0 [6.0-26.0] days, P < .001). There were no differences in time to ADAMTS-13 restoration from PEX start (28.0 [17.2-47.5], 27.0 [19.0-37.5] and 29.5 [15.2-45.0] days in early caplacizumab-treated, late caplacizumab-treated and non-caplacizumab-treated episodes). Early administered caplacizumab does not prevent the requirement for immunosuppression but has beneficial effects by shortening PEX requirement without major safety concerns.
卡泊珠单抗可阻止血管性血友病因子与血小板之间的相互作用，用于治疗免疫性血栓性血小板减少性紫癜（iTTP）。其使用与血浆置换（PEX）暂停后ADAMTS13活性恢复延迟有关。我们分析了西班牙血栓性血小板减少性紫癜登记处108例患者中113次iTTP发作的结果，其中75次发作接受了卡泊珠单抗治疗。卡泊珠单抗缩短了血小板计数恢复正常的时间，减少了PEX需求、病情加重和复发情况。在PEX结束后达到ADAMTS13活性≥20%的时间方面，接受卡泊珠单抗治疗的发作与未接受治疗的发作之间没有差异（中位数[四分位间距]，14.5[7.7 - 27.2]天对13.0[8.0 - 29.0]天，P = 0.653）。然而，考虑到36次在iTTP诊断后≤3天开始使用卡泊珠单抗的发作，从PEX结束时ADAMTS13恢复的时间高于在iTTP诊断后>3天开始使用卡泊珠单抗的发作（20.0[12.0 - 43.0]天对11.0[3.5 - 20.0]天，P = 0.003）或高于未接受卡泊珠单抗治疗的发作（P = 0.033）。这一发现可能与早期接受卡泊珠单抗治疗的发作中PEX持续时间明显短于晚期接受卡泊珠单抗治疗的发作（5.5[4.0 - 9.0]天对15.0[11.0 - 21.5]天，P < 0.001）或未接受卡泊珠单抗治疗的发作（11.0[6.0 - 26.0]天，P < 0.001）有关。从PEX开始时ADAMTS - 13恢复的时间在早期接受卡泊珠单抗治疗、晚期接受卡泊珠单抗治疗和未接受卡泊珠单抗治疗的发作中没有差异（分别为28.0[17.2 - 47.5]天、27.0[19.0 - 37.5]天和29.5[15.2 - 45.0]天）。早期使用卡泊珠单抗并不能避免免疫抑制的需求，但通过缩短PEX需求具有有益效果，且没有重大安全问题。
