Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain

BACKGROUND: Waldenström's macroglobulinemia (WM) is a B-cell neoplasia characterized by the infiltration of lymphoplasmacytic lymphoma cells in the bone marrow and abnormal secretion of IgM paraprotein. Ibrutinib, a Bruton tyrosine kinase inhibitor (BTKi), showed high efficacy in WM clinical tr...

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Autores: Fernández de Larrea, Carlos, Loscertales, Javier, Cabañas, Valentín, Freiria, Carmen, Blanchard, María Jesús, Askari, Elham, Lorenzo Pérez, Marcos, Abril, Laura, López Pardo, Jordi, Pinzón, Sergio, Díaz Gálvez, Javier, Amer Salas, Neus, Ramírez Páyer, Ángel, García Sánchez, Ricarda, Alcalá, María Magdalena, Capote, Francisco Javier, Moreno, David, Bolumburu, Celia, Rubio-Azpeitia, Eva, García Sanz, Ramón
Tipo de documento: artigo
Data de publicação:2025
País:España
Recursos:Conselleria de Salut i Consum del Govern de les Illes Balears
Repositório:Docusalut
Idioma:inglês
OAI Identifier:oai:docusalut.com:20.500.13003/26119
Acesso em linha:https://hdl.handle.net/20.500.13003/26119
Access Level:Acceso aberto
Palavra-chave:Spain
España
Waldenström’s macroglobulinemia
ibrutinib
real-world evidence
routine clinical practice
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spelling Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in SpainFernández de Larrea, CarlosLoscertales, JavierCabañas, ValentínFreiria, CarmenBlanchard, María JesúsAskari, ElhamLorenzo Pérez, MarcosAbril, LauraLópez Pardo, JordiPinzón, SergioDíaz Gálvez, JavierAmer Salas, NeusRamírez Páyer, ÁngelGarcía Sánchez, RicardaAlcalá, María MagdalenaCapote, Francisco JavierMoreno, DavidBolumburu, CeliaRubio-Azpeitia, EvaGarcía Sanz, RamónSpainEspañaSpainWaldenström’s macroglobulinemiaibrutinibreal-world evidenceroutine clinical practiceBACKGROUND: Waldenström's macroglobulinemia (WM) is a B-cell neoplasia characterized by the infiltration of lymphoplasmacytic lymphoma cells in the bone marrow and abnormal secretion of IgM paraprotein. Ibrutinib, a Bruton tyrosine kinase inhibitor (BTKi), showed high efficacy in WM clinical trials. However, there is limited real-world data regarding its effectiveness and safety in routine clinical practice. OBJECTIVES: The MACRO study aimed to investigate the clinical, genetic, and demographic characteristics of WM patients treated with ibrutinib-based therapies in Spain. Key secondary objectives included describing effectiveness and safety profile. DESIGN: Retrospective observational. METHODS: This multicenter, observational, retrospective study included adult patients diagnosed with symptomatic WM treated with ibrutinib since its commercial approval in Spain in 2016. Data were collected from 19 hospitals through retrospective medical chart reviews. RESULTS: Fifty-two eligible patients were recruited. The median age at the start of ibrutinib treatment was 74 years. Most of patients were male (65.4%) and had an Eastern Cooperative Oncology Group performance status of 0-1 (89.7%). Overall response rate was 92.2%, with a major response rate of 80.5%. Median progression-free survival (PFS) was 57.2 months, and the estimated 2-year overall survival rate was 89.2%. No significant differences in PFS were identified based on the parameters defining risk subgroups, nor did they vary according to treatment line, initial dose, or treatment schedule. Most common adverse events included bleeding (30.8%), diarrhea (23.1%), and infections (15.4%), with most of them being grades 1-2. No new safety signs were identified. CONCLUSION: This study presents real-world evidence on the characteristics and outcomes of WM patients treated with ibrutinib in Spain, showing it to be effective with a manageable safety profile consistent with clinical trial results. These findings support ibrutinib as a valuable treatment option for WM in real-world settings.20252025-10-0620252025-10-06research articlehttp://purl.org/coar/resource_type/c_2df8fbb1info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/20.500.13003/26119reponame:Docusalutinstname:Conselleria de Salut i Consum del Govern de les Illes BalearsInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:docusalut.com:20.500.13003/261192026-06-22T12:44:07Z
dc.title.none.fl_str_mv Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
title Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
spellingShingle Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
Fernández de Larrea, Carlos
Spain
España
Spain
Waldenström’s macroglobulinemia
ibrutinib
real-world evidence
routine clinical practice
title_short Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
title_full Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
title_fullStr Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
title_full_unstemmed Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
title_sort Observational retrospective study of the treatment of Waldenström's macroglobulinemia with ibrutinib in routine clinical practice in Spain
dc.creator.none.fl_str_mv Fernández de Larrea, Carlos
Loscertales, Javier
Cabañas, Valentín
Freiria, Carmen
Blanchard, María Jesús
Askari, Elham
Lorenzo Pérez, Marcos
Abril, Laura
López Pardo, Jordi
Pinzón, Sergio
Díaz Gálvez, Javier
Amer Salas, Neus
Ramírez Páyer, Ángel
García Sánchez, Ricarda
Alcalá, María Magdalena
Capote, Francisco Javier
Moreno, David
Bolumburu, Celia
Rubio-Azpeitia, Eva
García Sanz, Ramón
author Fernández de Larrea, Carlos
author_facet Fernández de Larrea, Carlos
Loscertales, Javier
Cabañas, Valentín
Freiria, Carmen
Blanchard, María Jesús
Askari, Elham
Lorenzo Pérez, Marcos
Abril, Laura
López Pardo, Jordi
Pinzón, Sergio
Díaz Gálvez, Javier
Amer Salas, Neus
Ramírez Páyer, Ángel
García Sánchez, Ricarda
Alcalá, María Magdalena
Capote, Francisco Javier
Moreno, David
Bolumburu, Celia
Rubio-Azpeitia, Eva
García Sanz, Ramón
author_role author
author2 Loscertales, Javier
Cabañas, Valentín
Freiria, Carmen
Blanchard, María Jesús
Askari, Elham
Lorenzo Pérez, Marcos
Abril, Laura
López Pardo, Jordi
Pinzón, Sergio
Díaz Gálvez, Javier
Amer Salas, Neus
Ramírez Páyer, Ángel
García Sánchez, Ricarda
Alcalá, María Magdalena
Capote, Francisco Javier
Moreno, David
Bolumburu, Celia
Rubio-Azpeitia, Eva
García Sanz, Ramón
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv
dc.subject.none.fl_str_mv Spain
España
Spain
Waldenström’s macroglobulinemia
ibrutinib
real-world evidence
routine clinical practice
topic Spain
España
Spain
Waldenström’s macroglobulinemia
ibrutinib
real-world evidence
routine clinical practice
description BACKGROUND: Waldenström's macroglobulinemia (WM) is a B-cell neoplasia characterized by the infiltration of lymphoplasmacytic lymphoma cells in the bone marrow and abnormal secretion of IgM paraprotein. Ibrutinib, a Bruton tyrosine kinase inhibitor (BTKi), showed high efficacy in WM clinical trials. However, there is limited real-world data regarding its effectiveness and safety in routine clinical practice. OBJECTIVES: The MACRO study aimed to investigate the clinical, genetic, and demographic characteristics of WM patients treated with ibrutinib-based therapies in Spain. Key secondary objectives included describing effectiveness and safety profile. DESIGN: Retrospective observational. METHODS: This multicenter, observational, retrospective study included adult patients diagnosed with symptomatic WM treated with ibrutinib since its commercial approval in Spain in 2016. Data were collected from 19 hospitals through retrospective medical chart reviews. RESULTS: Fifty-two eligible patients were recruited. The median age at the start of ibrutinib treatment was 74 years. Most of patients were male (65.4%) and had an Eastern Cooperative Oncology Group performance status of 0-1 (89.7%). Overall response rate was 92.2%, with a major response rate of 80.5%. Median progression-free survival (PFS) was 57.2 months, and the estimated 2-year overall survival rate was 89.2%. No significant differences in PFS were identified based on the parameters defining risk subgroups, nor did they vary according to treatment line, initial dose, or treatment schedule. Most common adverse events included bleeding (30.8%), diarrhea (23.1%), and infections (15.4%), with most of them being grades 1-2. No new safety signs were identified. CONCLUSION: This study presents real-world evidence on the characteristics and outcomes of WM patients treated with ibrutinib in Spain, showing it to be effective with a manageable safety profile consistent with clinical trial results. These findings support ibrutinib as a valuable treatment option for WM in real-world settings.
publishDate 2025
dc.date.none.fl_str_mv 2025
2025-10-06
2025
2025-10-06
dc.type.none.fl_str_mv research article
http://purl.org/coar/resource_type/c_2df8fbb1
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/20.500.13003/26119
url https://hdl.handle.net/20.500.13003/26119
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Docusalut
instname:Conselleria de Salut i Consum del Govern de les Illes Balears
instname_str Conselleria de Salut i Consum del Govern de les Illes Balears
reponame_str Docusalut
collection Docusalut
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15.812455

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