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, 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 |
بيانات النشر: | American Society of Hematology |
سنة النشر: | 2022 |
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. |
نوع الوثيقة: | article in journal/newspaper |
وصف الملف: | application/pdf |
اللغة: | English |
تدمد: | 2473-9537 |
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 |
DOI: | 10.1182/bloodadvances.2022008028 |
الإتاحة: | https://doi.org/10.1182/bloodadvances.2022008028Test https://hdl.handle.net/11351/9693Test |
حقوق: | Attribution-NonCommercial-NoDerivatives 4.0 International ; http://creativecommons.org/licenses/by-nc-nd/4.0Test/ ; info:eu-repo/semantics/openAccess |
رقم الانضمام: | edsbas.5E4262A7 |
قاعدة البيانات: | BASE |
تدمد: | 24739537 |
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DOI: | 10.1182/bloodadvances.2022008028 |