Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease

Authors: Rugeri, L; Harroche, A; Repessé, Y; Desprez, D; Petesch, BP; Chamouni, P; Biron, C; Frotscher, B; Catovic, H; Bracquart, D; Martin, C; Trossaërt, M; Meunier, S; D’Oiron, R

Affiliations : Unité Hémostase Clinique, Hospices Civils de Lyon, Bron, France. Hemophilia Care Centre, Hematology unit, Hôpital Universitaire Necker enfants malades, Paris, France. Unité Hémostase et Centre Régional de Traitement des maladies Hémorragiques, Institut de Biologie Clinique, Hôpital Charles Nicolle, Rouen, France. Centre de Traitement de l’Hémophilie, CHU Strasbourg, Strasbourg, France. Unité Hémostase Service hématologie, CHU Brest, Brest, France. Unité Hémostase et Centre Régional de Traitement des maladies Hémorragiques, Institut de Biologie Clinique, Hôpital Charles Nicolle, Rouen, France. Département d’Hématologie biologique, CHU de Montpellier, Montpellier, France. CRTH – Service d’hématologie biologique, CHU de Nancy, Nancy, France. CSL Behring, Paris, France.

Publication: European journal of haematology ; 2022 April

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.