Management of von Willebrand disease with a factor VIII‐poor von Willebrand factor concentrate: Results from a prospective observational post‐marketing study

Autor: Itzhar‐baïkian, Nathalie, Borel‐derlon, Annie, Goudemand, Jenny, Bridey, Françoise, Claeyssens, Segolene, Itzhar-Baikian, Nathalie, Harroche, Annie, Desprez, Dominique, Negrier, Claude, Chamouni, Pierre, Chambost, Hervé, Henriet, Céline, Susen, Sophie
Přispěvatelé: Hopital Saint-Louis [AP-HP] (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Hôpital Côte de Nacre [CHU Caen], CHU Caen, Normandie Université (NU)-Tumorothèque de Caen Basse-Normandie (TCBN)-Normandie Université (NU)-Tumorothèque de Caen Basse-Normandie (TCBN), Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Effilux, Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Service d'hématologie biologique, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Service d'immuno-hématologie pédiatrique [CHU Necker], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Necker - Enfants Malades [AP-HP], Département d'Oncologie et Hématologie [Strasbourg], Les Hôpitaux Universitaires de Strasbourg (HUS), Hôpital Edouard Herriot [CHU - HCL], Hospices Civils de Lyon (HCL), Laboratoire d'hématologie [Rouen], CHU Rouen, Normandie Université (NU)-Normandie Université (NU)-Université de Rouen Normandie (UNIROUEN), Normandie Université (NU), Assistance Publique - Hôpitaux de Marseille (APHM), Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), CHU Toulouse [Toulouse], Lucas, Nelly
Jazyk: angličtina
Rok vydání: 2020
Předmět:
[SDV.MHEP.HEM] Life Sciences [q-bio]/Human health and pathology/Hematology
medicine.medical_specialty
030204 cardiovascular system & hematology
von Willebrand factor
03 medical and health sciences
0302 clinical medicine
Von Willebrand factor
post‐marketing
Internal medicine
Breakthrough bleeding
hemic and lymphatic diseases
Von Willebrand disease
Humans
Medicine
Childbirth
Prospective Studies
post-marketing
Marketing
Thrombotic risk
biology
business.industry
HAEMOSTASIS
clinical trial
[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology
Hematology
Original Articles
medicine.disease
3. Good health
Clinical trial
von Willebrand Diseases
Tolerability
factor VIII
biology.protein
Female
Observational study
Original Article
France
medicine.symptom
business
von Willebrand disease
Zdroj: Journal of Thrombosis and Haemostasis
Journal of Thrombosis and Haemostasis, 2020, 18 (8), pp.1922-1933. ⟨10.1111/jth.14928⟩
Journal of Thrombosis and Haemostasis, Wiley, 2020, 18 (8), pp.1922-1933. ⟨10.1111/jth.14928⟩
ISSN: 1538-7933
1538-7836
DOI: 10.1111/jth.14928⟩
Popis: International audience; Background A triple-secured plasma-derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN(R), was approved in France in 2003, and then in other countries for the treatment of patients with von Willebrand disease (VWD). Objective To investigate long-term safety and efficacy of the product in real-life over the first 5 post-approval years. Patients/Methods This prospective, observational, national post-marketing study (PMS) enrolled patients of all ages and VWD types. Patients were observed for up to 3 years and treated for one or more occasions. Efficacy was assessed for each major event. Breakthrough bleeding rate 3 days post-infusion and annualized bleeding rate (ABR) were also evaluated for long-term prophylaxis. Results Overall, 155 of 174 patients enrolled from 31 centers were eligible for efficacy assessment. Most patients (76.8%) were severely affected (VWF:RCo = 12 months. Excellent tolerability was confirmed with no safety concerns. No thrombotic events were observed. Conclusions Results from this PMS increase the clinical experience of a FVIII-poor pdVWF in patients of all ages and VWD types including those with thrombotic risk factors and emphasize that giving FVIII is not always mandatory to effectively treat patients with severe VWD.
Databáze: OpenAIRE
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