Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura

• Published in: Blood advances Vol. 6; no. 24; pp. 6219 - 6227
• Main Authors: Izquierdo, Cristina Pascual, Mingot-Castellano, María Eva, Fuentes, Ana E. Kerguelen, García-Arroba Peinado, José, Cid, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez-Seguí, Inés, de la Rubia, Javier, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García-Muñoz, Nadia, Vara, Míriam, Zarzoso, Miguel Fernández, García-Candel, Faustino, Paciello, María Liz, García-García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Estévez, Julia Vidán, Jiménez, Gemma Moreno, López Lorenzo, José Luis, Arias, Elena González, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez-Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, del Rio-Garma, Julio
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 27-12-2022; The American Society of Hematology
• Subjects: Adult; Humans; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombotic Thrombocytopenic - drug therapy; Regular; Retrospective Studies; Rituximab - adverse effects; Standard of Care; Thrombosis
• DOI: 10.1182/bloodadvances.2022008028
• EISSN: 2473-9537
• ISSN: 2473-9529

•Caplacizumab reduces exacerbation and refractoriness in iTTP.•As initial therapy, caplacizumab accelerates response and reduces the need for PEX and hospital stay.
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Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with standard of care regimens. When administered within the first 3 days after PEX, it also provided a faster clinical response, reducing hospitalization time and the need for PEX.