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

Abstract

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.