Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease

Abstract

Background The efficacy and safety of wilate (human von Willebrand factor/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD. Objectives The objectives of this observational, prospective, phase 4 study were to evaluate the safety, tolerability, and effectiveness of wilate in on-demand treatment of bleeding episodes (BEs), long-term prophylaxis, and surgical prophylaxis among patients with any type of VWD. Methods Patients were enrolled at 31 study centers in 11 countries and followed for up to 2 years. Safety endpoints included adverse drug reactions (ADRs) and drug tolerability. Effectiveness was assessed using annualized bleeding rates (ABRs) during prophylaxis and predefined criteria for the treatment of BEs and surgical prophylaxis. Results A total of 111 patients (76 [68%] female) including 41 (37%) children were treated with wilate. Twenty-five patients received prophylaxis, 29 on-demand treatment, and 62 surgical prophylaxis. Tolerability was rated by patients as “excellent” for 96.2% of 6,497 infusions. No unexpected ADRs or thrombotic events were reported. Median ABR during prophylaxis was 1.9. Effectiveness was assessed as “excellent” or “good” by patients and investigators for 100% of BEs treated on-demand, 98% (patient rating) and 99% (investigator rating) of breakthrough BEs, and 99% of surgical procedures (investigator rating). Conclusion wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.

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

“…A search in the medical literature has revealed that among the 13 studies showing a benefit of the prophylaxis regimen, only 7 are prospectively designed, and they all suffer from a large heterogeneity in study design, study population, VWD subtypes, and use of type of concentrate (Table 5). 5,12,[14][15][16][17][18][19][20][21][22][23][24] Most patients included in the study were type 3 patients, reflecting the severity of the clinical forms leading the clinicians to broadly initiate a prophylaxis regimen. The number of type 2B patients requiring prophylaxis was also high, due to their low baseline platelet count.…”

“…A search in the medical literature has revealed that among the 13 studies showing a benefit of the prophylaxis regimen, only 7 are prospectively designed, and they all suffer from a large heterogeneity in study design, study population, VWD subtypes, and use of type of concentrate (Table 5). 5,12,14–24 …”

Management of a patient with acute myeloid leukaemia with a diagnosis of type 2 von Willebrand disease and a novel variant within the von Willebrand factor (VWF) gene

“…9 The ISTH guidelines recommend 3 months of therapeutic anticoagulation if the line is to remain in situ but in the setting of thrombocytopenia, the decision to anticoagulate needs to be made balancing the risks of clot propagation/ embolisation and bleeding. 10 Sholzberg et al 11 demonstrated the safety of Wilate® when no thromboembolic events were noted in patients on a prophylactic regimen. However, an older study looking at specific cases of VTE in patients with VWD concluded that it is biologically possible for patients receiving repeated FVIII:VWF infusions with pro-thrombotic risk factors to develop VTE.…”