المؤلفون: Pascual Izquierdo, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana E., García-Arroba Peinado, José, Cid, Joan, Moraima Jimenez, Maria, 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, Fernández Zarzoso, Miguel, García Candel, Faustino, Paciello, María Liz, García García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Vidán Estévez, Julia, Moreno Jiménez, Gemma, López Lorenzo, José Luis, González Arias, Elena, 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

المصدر: Blood Advances. Vol. 6, nº 24, December 2022, pp. 6219 - 6227

مصطلحات موضوعية: caplacizumab, prednisone, rituximab

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. ; 9 páginas

وصف الملف: application/pdf

العلاقة: http://hdl.handle.net/10498/29789Test

الإتاحة: https://doi.org/10.1182/bloodadvances.2022008028Test
http://hdl.handle.net/10498/29789Test

المؤلفون: Pascual Izquierdo, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana E., García Arroba Peinado, José, Cid Vidal, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez Seguí, Inés, Rubia, Javier de la, Martin, 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, Vidán Estévez, Julia, Moreno Jiménez, Gemma, López Lorenzo, José Luis, González Arias, Elena, 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, Rio Garma, Julio del, Spanish Apheresis Group (GEA), Spanish Thrombotic Thrombocytopenic Purpura Registry (REPTT)

المصدر: Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))

مصطلحات موضوعية: Rituximab, Trombosi, Assaigs clínics, Thrombosis, Clinical trials

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.

وصف الملف: 9 p.; application/pdf

العلاقة: Reproducció del document publicat a: https://doi.org/10.1182/bloodadvances.2022008028Test; Blood Advances, 2022, vol. 6, num. 24, p. 6219-6227; https://doi.org/10.1182/bloodadvances.2022008028Test; http://hdl.handle.net/2445/196688Test

الإتاحة: https://doi.org/10.1182/bloodadvances.2022008028Test
http://hdl.handle.net/2445/196688Test

المؤلفون: Pascual, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana Esther, García-Arroba Peinado, José, Cid, Joan, Jiménez, Moraima, VALCARCEL, DAVID

المساهمون: Institut Català de la Salut, Izquierdo CP Department of Hematology, Hospital General Universitario Gregorio Marañon (HGUGM) Madrid, Instituto de Investigación Gregorio Marañon, Madrid, Spain. Mingot-Castellano ME Hematology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla, Seville, Spain. Fuentes AEK Department of Hematology and Hemotherapy, Hospital La Paz, Madrid, Spain. García-Arroba Peinado J Banc de Sang i Teixits, Hospital Universitario Joan XXIII, Tarragona, Spain. Cid J Apheresis and Cellular Therapy Unit, Department of Hemotherapy & Hemostasis, ICMHO, IDIBAPS, Hospital Clínic de Barcelona, Barcelona, Spain. Jimenez MM, Valcarcel D Servei d’Hematologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Experimental Hematology, Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain, Vall d'Hebron Barcelona Hospital Campus

المصدر: Scientia

مصطلحات موضوعية: Púrpura trombocitopènica trombòtica - Tractament, Avaluació de resultats (Assistència sanitària), DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Blood Coagulation Disorders::Purpura::Purpura, Thrombocytopenic::Purpura, Thrombotic Thrombocytopenic, Other subheadings::Other subheadings::Other subheadings::/drug therapy, ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome, ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos de la coagulación sanguínea::púrpura::púrpura trombocitopénica::púrpura trombocitopénica trombótica, Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia, TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento

الوصف: Effectiveness; Caplacizumab; Immune thrombotic thrombocytopenic purpura ; Eficàcia; Caplacizumab; Púrpura trombocitopènica trombòtica immunitària ; Eficacia; Caplacizumab; Púrpura trombocitopénica trombótica inmunitaria ; 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.

وصف الملف: application/pdf

العلاقة: Blood Advances;6(24); https://doi.org/10.1182/bloodadvances.2022008028Test; Izquierdo CP, Mingot-Castellano ME, Kerguelen Fuentes AE, Peinado JGA, Cid J, Jimenez MM, et al. Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura. Blood Adv. 2022 Dec 27;6(24):6219–27.; https://hdl.handle.net/11351/9693Test; 000923057000001

الإتاحة: https://doi.org/10.1182/bloodadvances.2022008028Test
https://hdl.handle.net/11351/9693Test

المؤلفون: Gómez-Seguí, Inés, Francés Aracil, E., Mingot-Castellano, María Eva, Vara Pampliega, M., Goterris Viciedo, R., García-Candel, Faustino, Pascual-Izquierdo, Cristina, Río-Garma, Julio del, Guerra Domínguez, L., Vicuña Andrés, I., García-Arroba Peinado, José, Zalba, Saioa, Vidan Estévez, J. M., González Arias, E., Campuzano Saavedra, V., García Gala, J. M., Ortega Sánchez, S., Martínez Nieto, J., Pardo Gambarte, L., Solé Rodríguez, M., Fernández-Docampo, M., Ávila Idrovo, L. F., Hernández, L., Cid, Joan, Rubia, Javier de la, from the Spanish TTP Registry (REPTT, Registro Español de la Purpura Trombocitopénica Trombótica)

مصطلحات موضوعية: Aged, Caplacizumab, Rituximab, Thrombotic thrombocytopenic purpura

الوصف: Immune thrombotic thrombocytopenic purpura (iTTP) is an ultra-rare disease that seldom occurs in the elderly. Few reports have studied the clinical course of iTTP in older patients. In this study, we have analysed the clinical characteristics at presentation and response to therapy in a series of 44 patients with iTTP ≥60 years at diagnosis from the Spanish TTP Registry and compared them with 209 patients with <60 years at diagnosis from the same Registry. Similar symptoms and laboratory results were described in both groups, except for a higher incidence of renal dysfunction among older patients (23% vs. 43.1%; p = 0.008). Front-line treatment in patients ≥60 years was like that administered in younger patients. Also, no evidence of a difference in clinical response and overall survival was seen in both groups. Of note, 14 and 25 patients ≥60 years received treatment with caplacizumab and rituximab, respectively, showing a favourable safety and efficacy profile, like that observed in patients <60 years. ; Peer reviewed

العلاقة: The underlying dataset has been published as supplementary material of the article in the publisher platform at https://doi.org/10.1111/bjh.19109Test; https://doi.org/10.1111/bjh.19109Test; Sí; British Journal of Haematology 203(5): 860-871 (2023); http://hdl.handle.net/10261/350868Test

الإتاحة: https://doi.org/10.1111/bjh.19109Test
http://hdl.handle.net/10261/350868Test

المؤلفون: 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

المصدر: Blood Advances; December 2022, Vol. 6 Issue: 24 p6219-6227, 9p

مستخلص: 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.

المؤلفون: Mingot, Maria Eva, Pascual, Cristina Izquierdo, Garcia-Candel, Faustino, Martínez Nieto, Jorge, García-Arroba Peinado, José, De La Rubia, Javier, Gomez Segui, Ines, Pacielo Coronel, Maria Liz, Valcárcel, David, Jiménez Balarezo, Moraima, Cid, Joan, Lozano, Miquel, Garcia Gala, Jose Maria, Angos Vazquez, Sonia, Vara, Míriam, Guerra, Luisa, Avila Idrobo, Francisca, Oliva Hernandez, ANA Yurena, Zalba, Saoia, Tallon Ruiz, Inmaculada, Ortega Sánchez, Sandra, Goterris, Rosa, Moreno Jimenez, Maria, Dominguez Acosta, Lourdes, Arraiz Ramirez, Maria, Hernández, Luis, Flores Ballesteros, Elena, Del Rio Garma, Julio

المصدر: Blood; November 2023, Vol. 142 Issue: 1, Number 1 Supplement 1 p5394-5394, 1p

مستخلص: INTRODUCTION