Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study

IntroductionPlasma-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...

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Autores: Benítez Hidalgo, Olga, Marzo Alonso, María Cristina, López Jaime, Francisco José, Carrasco, Marina, Nieto Hernández, Maria Del Mar, Afonso, Natalia, Lei, Alejhandra, Whyms, Dermot, Hanna, Kim, Aragonés, Maria Esperança, Torres, Mireia, Oliveras, Juan
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Institución:Universitat de Lleida (UdL)
Repositorio:Repositori Obert UdL
OAI Identifier:oai:repositori.udl.cat:10459.1/468794
Acceso en línea:https://doi.org/10.1177/1076029625132759
https://hdl.handle.net/10459.1/468794
Access Level:acceso abierto
Palabra clave: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 Disease: A Prospective, Observational, Post-Authorization StudyBenítez Hidalgo, OlgaMarzo Alonso, María CristinaLópez Jaime, Francisco JoséCarrasco, MarinaNieto 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 diseaseIntroductionPlasma-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.MethodsThis 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.ResultsFifteen 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.ConclusionsThis 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.SAGE Publications2025info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttps://doi.org/10.1177/1076029625132759https://hdl.handle.net/10459.1/468794reponame:Repositori Obert UdL instname:Universitat de Lleida (UdL)InglésReproducció del document publicat a: https://doi.org/10.1177/1076029625132759Clinical and Applied Thrombosis/Hemostasis, 2025, vol. 31cc-by-nc (c) The Authors, 2025Attribution-NonCommercial 4.0 Internationalinfo:eu-repo/semantics/openAccesshttp://creativecommons.org/licenses/by-nc/4.0/oai:repositori.udl.cat:10459.1/4687942026-06-24T12:42:17Z
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: A Prospective, Observational, Post-Authorization Study
spellingShingle Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study
Benítez Hidalgo, Olga
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: A Prospective, Observational, Post-Authorization Study
title_full Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study
title_fullStr Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study
title_full_unstemmed Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study
title_sort Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study
dc.creator.none.fl_str_mv Benítez Hidalgo, Olga
Marzo Alonso, María Cristina
López Jaime, Francisco José
Carrasco, Marina
Nieto Hernández, Maria Del Mar
Afonso, Natalia
Lei, Alejhandra
Whyms, Dermot
Hanna, Kim
Aragonés, Maria Esperança
Torres, Mireia
Oliveras, Juan
author Benítez Hidalgo, Olga
author_facet Benítez Hidalgo, Olga
Marzo Alonso, María Cristina
López Jaime, Francisco José
Carrasco, Marina
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, María Cristina
López Jaime, Francisco José
Carrasco, Marina
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.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 IntroductionPlasma-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.MethodsThis 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.ResultsFifteen 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.ConclusionsThis 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 2025
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://doi.org/10.1177/1076029625132759
https://hdl.handle.net/10459.1/468794
url https://doi.org/10.1177/1076029625132759
https://hdl.handle.net/10459.1/468794
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Reproducció del document publicat a: https://doi.org/10.1177/1076029625132759
Clinical and Applied Thrombosis/Hemostasis, 2025, vol. 31
dc.rights.none.fl_str_mv cc-by-nc (c) The Authors, 2025
Attribution-NonCommercial 4.0 International
info:eu-repo/semantics/openAccess
http://creativecommons.org/licenses/by-nc/4.0/
rights_invalid_str_mv cc-by-nc (c) The Authors, 2025
Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv SAGE Publications
publisher.none.fl_str_mv SAGE Publications
dc.source.none.fl_str_mv reponame:Repositori Obert UdL
instname:Universitat de Lleida (UdL)
instname_str Universitat de Lleida (UdL)
reponame_str Repositori Obert UdL
collection Repositori Obert UdL
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15.812429

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