Pharmacokinetics, efficacy, and safety of a plasma-derived VWF/FVIII concentrate (VONCENTO) for on-demand and prophylactic treatment in patients with von Willebrand disease (SWIFT-VWD study)

Abstract

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<p>An Open-Label Extension Study to Assess the Long-Term Efficacy and Safety of a Plasma-Derived von Willebrand Factor (VWF)/Factor VIII (FVIII) Concentrate in Patients with von Willebrand Disease (SWIFT-VWDext Study)</p>

“…The median number of NSB events per year in the prophylaxis arm (children: 22.2 events; adults: 0.6 events) was similar to the median reported in the prophylaxis arms of the SWIFTLY-VWD pediatric and SWIFT-VWD adult/adolescent studies (23.5 and 1.0, respectively). 8 , 10 In patients treated on-demand, the median number of bleeding events per year in this study (12.71 events) was lower than in the on-demand arm of SWIFT-VWD (19.5 events). 8 This would be expected, as patients who experienced a higher rate of bleeding during the on-demand portion of the SWIFT-VWD study would have transitioned to prophylactic treatment in the second stage of that study and would have remained on prophylaxis during this extension study.…”

“…Nine (90.0%) patients in the prophylaxis arm reported a total of 118 NSB events, 96 of which required treatment. The median (Q1; Q3) number of NSB events per patient was 8.0 (1)(2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18)(19). Seven patients experienced a total of 12…”

“…7 Plasma-derived VWF/FVIII concentrate (pdVWF/FVIII; VONCENTO ® , CSL Behring, Germany) is a highconcentration, low-volume, high-purity concentrate, which contains a large proportion of high-molecular-weight multimers and a VWF/FVIII ratio of ~2.4:1. 8 pdVWF/FVIII is licensed by the European Medicines Agency (EMA) for the prevention and treatment of hemorrhage or surgical bleeding in adult, adolescent and pediatric patients with VWD. 9 The SWIFT ("Studies with von Willebrand factor/ Factor VIII") program evaluated the hemostatic efficacy and safety of pdVWF/FVIII in pediatric (0 to <12 years of age; SWIFTLY-VWD study), or adolescent and adult (≥12 years of age; SWIFT-VWD) patients with VWD type 1, 2A, or 3 in whom treatment with a VWF product was required for prophylactic therapy, hemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.…”

“…Plasma-derived VWF/FVIII concentrate (pdVWF/FVIII; VONCENTO ® , CSL Behring, Germany) is a high-concentration, low-volume, high-purity concentrate, which contains a large proportion of high-molecular-weight multimers and a VWF/FVIII ratio of ~2.4:1. 8 pdVWF/FVIII is licensed by the European Medicines Agency (EMA) for the prevention and treatment of hemorrhage or surgical bleeding in adult, adolescent and pediatric patients with VWD. 9…”

“…The SWIFT (“Studies with von Willebrand factor/Factor VIII”) program evaluated the hemostatic efficacy and safety of pdVWF/FVIII in pediatric (0 to <12 years of age; SWIFTLY-VWD study), or adolescent and adult (≥12 years of age; SWIFT-VWD) patients with VWD type 1, 2A, or 3 in whom treatment with a VWF product was required for prophylactic therapy, hemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event. 8 , 10 Following the completion of these studies, patients who had participated in either SWIFTLY-VWD or SWIFT-VWD were able to transition to an extension study (SWIFT-VWDext). The aim of this study was to build on the results of the previous studies to assess the long-term efficacy and safety of pdVWF/FVIII in pediatric, adolescent, and adult patients with VWD and to assist the clinical understanding regarding the use of on-demand treatment versus set prophylaxis treatment with a VWF product.…”