Effectiveness of long‐term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease. (29th April 2022)

• Record Type: Journal Article
• Authors: Rugeri, Lucia; Harroche, Annie; Repessé, Yohan; Desprez, Dominique; Petesch, Brigitte Pan; Chamouni, Pierre; Biron, Christine; Frotscher, Birgit; Catovic, Hasan; Bracquart, Diane; Martin, Cédric; Trossaërt, Marc; Meunier, Sandrine; d'Oiron, Roseline
• Is Part Of: European journal of haematology. Volume 109:Number 1(2022)
• Page Start: 109
• Page End: 117
• Publication Date: 2022-04-29
• Subjects: bleeding -- FVIII/VWF concentrates -- prophylaxis -- von Willebrand disease -- Voncento®; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; Blood -- Periodicals; 616.15005
• DOI: 10.1111/ejh.13778
• Languages: English
• ISSNs: 0902-4441

Abstract

Abstract: Background: Patients with symptomatic von Willebrand disease (VWD) should be offered long‐term prophylaxis (LTP) to prevent recurrent bleedings. Our objective was to evaluate the effectiveness and safety of Voncento®, a plasma‐derived FVIII/VWF concentrate (ratio 1:2.4), administrated in LTP. Methods: We included patients from the OPALE study (May 2016 to April 2021), a French multicenter observational study following patients with inherited VWD, who received a Voncento® LTP during the study period. Results: Among the 130 OPALE‐study patients, 23 patients (12 women) received a LTP and were therefore included. The median (range) age was 16 (1–85) years; 16 patients were type 3, 1 was type 2A, 6 were type 2B. Before inclusion, 19 (83%) were under LTP and 4 (17%) received on‐demand (OD) treatment. The indications for initiating prophylaxis in the overall population were joint bleeding (43%), ear, nose, and throat (ENT) bleeding including epistaxis or oral bleeding (39%), and recurrent muscle hematoma (22%). The medians (ranges) dose of Voncento® per infusion, frequency, and weekly dose were 45 (33–109) IU/kg, 2 infusions per week, and 96 (44–222) IU/kg/week, respectively. The median (range) annualized bleeding rate (ABR) was 0.8, 0.7 (0–3.5), and 0 (0–2.3) for type 2A, 2B, 3 patients, respectively. There was no difference regarding to the dose, frequency of infusion, or in terms of ABR in 9/19 patients who replaced previous concentrates with Voncento®. During the study period, no adverse event was reported. Conclusion: These results suggest that Voncento® is effective to prevent recurrent bleedings in patients symptomatic VWD.