دورية أكاديمية
Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
| العنوان: | Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura |
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| المؤلفون: | Pascual-Izquierdo, Cristina, Mingot-Castellano, María Eva, Fuentes, Ana, García-Arroba Peinado, José, Cid, Joan, Jiménez, Moraima, Valcárcel, David, Gómez-Seguí, Inés, Rubia, Javier de la, Martín, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García-Muñoz, Nadia, Vara, Míriam, Fernández Zarzoso, Miguel, García-Candel, Faustino, Paciello, María Liz, García-García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Estévez, Julia Vidán, Moreno Jiménez, Gemma, López Lorenzo, José Luis, González-Arias, Candela, 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, Río-Garma, Julio del |
| بيانات النشر: | American Society of Hematology |
| سنة النشر: | 2022 |
| المجموعة: | Digital.CSIC (Consejo Superior de Investigaciones Científicas / Spanish National Research Council) |
| الوصف: | 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. ; Peer reviewed |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| ردمك: | 978-85-14-16692-4 85-14-16692-1 |
| تدمد: | 24739529 |
| العلاقة: | Publisher's version; The underlying dataset has been published as supplementary material of the article in the publisher platform at DOI 10.1182/bloodadvances.2022008028; https://doi.org/10.1182/bloodadvances.2022008028Test; Sí; Blood Advances 6(24): 6219-6227 (2022); http://hdl.handle.net/10261/307000Test; 2-s2.0-85141669213; https://api.elsevier.com/content/abstract/scopus_id/85141669213Test |
| DOI: | 10.1182/bloodadvances.2022008028 |
| الإتاحة: | https://doi.org/10.1182/bloodadvances.2022008028Test http://hdl.handle.net/10261/307000Test https://api.elsevier.com/content/abstract/scopus_id/85141669213Test |
| حقوق: | open |
| رقم الانضمام: | edsbas.C83BF6C9 |
| قاعدة البيانات: | BASE |
Full Text Finder | ردمك: | 9788514166924 8514166921 |
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| تدمد: | 24739529 |
| DOI: | 10.1182/bloodadvances.2022008028 |