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

Effectiveness; Caplacizumab; Immune thrombotic thrombocytopenic purpura

Detalles Bibliográficos
Autores: Pascual, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana Esther, García-Arroba Peinado, José, Cid, Joan, Jiménez, Moraima, VALCARCEL, DAVID
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2022
País:España
Institución:Departament de Salut de la Generalitat de Catalunya (DS)
Repositorio:Scientia. Dipòsit d'Informació Digital del Departament de Salut
OAI Identifier:oai:scientiasalut.gencat.cat:11351/9693
Acceso en línea:https://hdl.handle.net/11351/9693
Access Level:acceso abierto
Palabra clave: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
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dc.title.none.fl_str_mv Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
title Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
spellingShingle Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
Pascual, Cristina
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
title_short Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
title_full Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
title_fullStr Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
title_full_unstemmed Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
title_sort Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura
dc.creator.none.fl_str_mv Pascual, Cristina
Mingot Castellano, María Eva
Kerguelen Fuentes, Ana Esther
García-Arroba Peinado, José
Cid, Joan
Jiménez, Moraima
VALCARCEL, DAVID
author Pascual, Cristina
author_facet Pascual, Cristina
Mingot Castellano, María Eva
Kerguelen Fuentes, Ana Esther
García-Arroba Peinado, José
Cid, Joan
Jiménez, Moraima
VALCARCEL, DAVID
author_role author
author2 Mingot Castellano, María Eva
Kerguelen Fuentes, Ana Esther
García-Arroba Peinado, José
Cid, Joan
Jiménez, Moraima
VALCARCEL, DAVID
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv 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
dc.subject.none.fl_str_mv 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
topic 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
description Effectiveness; Caplacizumab; Immune thrombotic thrombocytopenic purpura
publishDate 2022
dc.date.none.fl_str_mv 2022
2023
2023
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/11351/9693
url https://hdl.handle.net/11351/9693
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Blood Advances;6(24)
https://doi.org/10.1182/bloodadvances.2022008028
dc.rights.none.fl_str_mv Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv American Society of Hematology
publisher.none.fl_str_mv American Society of Hematology
dc.source.none.fl_str_mv Scientia
reponame:Scientia. Dipòsit d'Informació Digital del Departament de Salut
instname:Departament de Salut de la Generalitat de Catalunya (DS)
instname_str Departament de Salut de la Generalitat de Catalunya (DS)
reponame_str Scientia. Dipòsit d'Informació Digital del Departament de Salut
collection Scientia. Dipòsit d'Informació Digital del Departament de Salut
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869418664828076032
spelling Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpuraPascual, CristinaMingot Castellano, María EvaKerguelen Fuentes, Ana EstherGarcía-Arroba Peinado, JoséCid, JoanJiménez, MoraimaVALCARCEL, DAVIDPúrpura trombocitopènica trombòtica - TractamentAvaluació de resultats (Assistència sanitària)DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Blood Coagulation Disorders::Purpura::Purpura, Thrombocytopenic::Purpura, Thrombotic ThrombocytopenicOther subheadings::Other subheadings::Other subheadings::/drug therapyANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment OutcomeENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos de la coagulación sanguínea::púrpura::púrpura trombocitopénica::púrpura trombocitopénica trombóticaOtros calificadores::Otros calificadores::Otros calificadores::/farmacoterapiaTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamientoEffectiveness; Caplacizumab; Immune thrombotic thrombocytopenic purpuraEficàcia; Caplacizumab; Púrpura trombocitopènica trombòtica immunitàriaEficacia; Caplacizumab; Púrpura trombocitopénica trombótica inmunitariaImmune 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.American Society of HematologyInstitut 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, SpainVall d'Hebron Barcelona Hospital Campus202320232022info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/9693Scientiareponame:Scientia. Dipòsit d'Informació Digital del Departament de Salutinstname:Departament de Salut de la Generalitat de Catalunya (DS)InglésBlood Advances;6(24)https://doi.org/10.1182/bloodadvances.2022008028Attribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:scientiasalut.gencat.cat:11351/96932026-06-12T09:38:37Z
score 15.812455

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