Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease

Introduction: Plasma-derived von Willebrand factor containing FVIII concentrates (pdVWF/FVIII-C) are indicated as replacement therapy for patients with von Willebrand disease (VWD). This study assessed safety and efficacy associated with long-term real-world experience of the pdVWF/FVIII-C, Fanhdi,...

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Autores: Benítez Hidalgo, Olga|||0000-0002-2883-9049, Marzo Alonso, Cristina, López Jaime, Francisco José, Carrasco Exposito, Marina|||0000-0001-8301-8000, Nieto Hernández, Maria del Mar, Afonso, Natalia, Lei, Alejhandra, Whyms, Dermot, Hanna, Kim, Aragonés, Maria Esperança, Torres, Mireia, Oliveras, Juan
Tipo de documento: artigo
Data de publicação:2025
País:España
Recursos:Universitat Autònoma de Barcelona
Repositório:Dipòsit Digital de Documents de la UAB
Idioma:inglês
OAI Identifier:oai:ddd.uab.cat:320040
Acesso em linha:https://ddd.uab.cat/record/320040
https://dx.doi.org/urn:doi:10.1177/10760296251327593
Access Level:Acceso aberto
Palavra-chave:Bleeding
Invasive procedures
On-demand treatment
Plasma-derived von Willebrand factor/factor VIII concentrate
Prophylaxis treatment
Real world evidence
Surgery
Von Willebrand disease
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spelling Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand DiseaseA Prospective, Observational, Post-Authorization StudyBenítez Hidalgo, Olga|||0000-0002-2883-9049Marzo Alonso, CristinaLópez Jaime, Francisco JoséCarrasco Exposito, Marina|||0000-0001-8301-8000Nieto Hernández, Maria del MarAfonso, NataliaLei, AlejhandraWhyms, DermotHanna, KimAragonés, Maria EsperançaTorres, MireiaOliveras, JuanBleedingInvasive proceduresOn-demand treatmentPlasma-derived von Willebrand factor/factor VIII concentrateProphylaxis treatmentReal world evidenceSurgeryVon Willebrand diseaseIntroduction: Plasma-derived von Willebrand factor containing FVIII concentrates (pdVWF/FVIII-C) are indicated as replacement therapy for patients with von Willebrand disease (VWD). This study assessed safety and efficacy associated with long-term real-world experience of the pdVWF/FVIII-C, Fanhdi, in patients with VWD. Methods: This observational, prospective, post-authorization cohort study was conducted at five centers in Spain. Patients with VWD were treated with the pdVWF/FVIII-C to achieve satisfactory hemostasis for on-demand (bleeding episodes and surgical/invasive procedures) and prophylaxis treatment. Clinical efficacy was evaluated as the response to treatment in both settings. Safety parameters were assessed. Results: Fifteen VWD patients received at least one dose of the pdVWF/FVIII-C and were followed for 12 months. Forty-six bleeding episodes were reported for 9 (60.0%) patients, and 6 surgical/invasive procedures for 5 (33.3%) patients. Most frequently reported bleedings were gastrointestinal (3 [33.0%] patients) and gynecological (3 [33.0%] patients). No complications nor bleeding episodes related to surgical/invasive procedures were reported. Overall clinical efficacy of treatment (including on-demand and prophylaxis) achieved 100% excellent and/or good (n = 15 patients), being excellent for 7 (46.7%) patients. There were 27 treatment-emergent adverse events in 8 (53.3%) patients, 11 serious adverse events in 3 (20.0%) patients, but none of them were drug-related. No clinical signs and symptoms of immunogenicity or thromboembolic events were reported. Conclusions: This real-world evidence study confirmed the efficacy of the pdVWF/FVIII-C as on-demand and/or prophylaxis treatment in patients with bleeding episodes or surgical procedures in VWD. Fanhdi was well tolerated without any safety concerns.Universitat Autònoma de Barcelona 22025-01-0120252025-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/320040https://dx.doi.org/urn:doi:10.1177/10760296251327593reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.https://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:3200402026-06-06T12:50:31Z
dc.title.none.fl_str_mv Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
A Prospective, Observational, Post-Authorization Study
title Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
spellingShingle Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
Benítez Hidalgo, Olga|||0000-0002-2883-9049
Bleeding
Invasive procedures
On-demand treatment
Plasma-derived von Willebrand factor/factor VIII concentrate
Prophylaxis treatment
Real world evidence
Surgery
Von Willebrand disease
title_short Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
title_full Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
title_fullStr Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
title_full_unstemmed Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
title_sort Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease
dc.creator.none.fl_str_mv Benítez Hidalgo, Olga|||0000-0002-2883-9049
Marzo Alonso, Cristina
López Jaime, Francisco José
Carrasco Exposito, Marina|||0000-0001-8301-8000
Nieto Hernández, Maria del Mar
Afonso, Natalia
Lei, Alejhandra
Whyms, Dermot
Hanna, Kim
Aragonés, Maria Esperança
Torres, Mireia
Oliveras, Juan
author Benítez Hidalgo, Olga|||0000-0002-2883-9049
author_facet Benítez Hidalgo, Olga|||0000-0002-2883-9049
Marzo Alonso, Cristina
López Jaime, Francisco José
Carrasco Exposito, Marina|||0000-0001-8301-8000
Nieto Hernández, Maria del Mar
Afonso, Natalia
Lei, Alejhandra
Whyms, Dermot
Hanna, Kim
Aragonés, Maria Esperança
Torres, Mireia
Oliveras, Juan
author_role author
author2 Marzo Alonso, Cristina
López Jaime, Francisco José
Carrasco Exposito, Marina|||0000-0001-8301-8000
Nieto Hernández, Maria del Mar
Afonso, Natalia
Lei, Alejhandra
Whyms, Dermot
Hanna, Kim
Aragonés, Maria Esperança
Torres, Mireia
Oliveras, Juan
author2_role author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universitat Autònoma de Barcelona
dc.subject.none.fl_str_mv Bleeding
Invasive procedures
On-demand treatment
Plasma-derived von Willebrand factor/factor VIII concentrate
Prophylaxis treatment
Real world evidence
Surgery
Von Willebrand disease
topic Bleeding
Invasive procedures
On-demand treatment
Plasma-derived von Willebrand factor/factor VIII concentrate
Prophylaxis treatment
Real world evidence
Surgery
Von Willebrand disease
description Introduction: Plasma-derived von Willebrand factor containing FVIII concentrates (pdVWF/FVIII-C) are indicated as replacement therapy for patients with von Willebrand disease (VWD). This study assessed safety and efficacy associated with long-term real-world experience of the pdVWF/FVIII-C, Fanhdi, in patients with VWD. Methods: This observational, prospective, post-authorization cohort study was conducted at five centers in Spain. Patients with VWD were treated with the pdVWF/FVIII-C to achieve satisfactory hemostasis for on-demand (bleeding episodes and surgical/invasive procedures) and prophylaxis treatment. Clinical efficacy was evaluated as the response to treatment in both settings. Safety parameters were assessed. Results: Fifteen VWD patients received at least one dose of the pdVWF/FVIII-C and were followed for 12 months. Forty-six bleeding episodes were reported for 9 (60.0%) patients, and 6 surgical/invasive procedures for 5 (33.3%) patients. Most frequently reported bleedings were gastrointestinal (3 [33.0%] patients) and gynecological (3 [33.0%] patients). No complications nor bleeding episodes related to surgical/invasive procedures were reported. Overall clinical efficacy of treatment (including on-demand and prophylaxis) achieved 100% excellent and/or good (n = 15 patients), being excellent for 7 (46.7%) patients. There were 27 treatment-emergent adverse events in 8 (53.3%) patients, 11 serious adverse events in 3 (20.0%) patients, but none of them were drug-related. No clinical signs and symptoms of immunogenicity or thromboembolic events were reported. Conclusions: This real-world evidence study confirmed the efficacy of the pdVWF/FVIII-C as on-demand and/or prophylaxis treatment in patients with bleeding episodes or surgical procedures in VWD. Fanhdi was well tolerated without any safety concerns.
publishDate 2025
dc.date.none.fl_str_mv 2
2025-01-01
2025
2025-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/320040
https://dx.doi.org/urn:doi:10.1177/10760296251327593
url https://ddd.uab.cat/record/320040
https://dx.doi.org/urn:doi:10.1177/10760296251327593
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
https://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
https://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:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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