Human plasma-derived FVIII/VWD concentrate (Biostate): a review of experimental and clinical pharmacokinetic, efficacy and safety data

Abstract

Human plasma-derived factor VIII/von Willebrand factor complex concentrates are used to control bleeding in patients with von Willebrand disease (VWD) or haemophilia A (HA). The properties of these haemostatic factor concentrates vary widely, which can have significant clinical implications.This review provides an extensive overview of the molecular properties, in addition to pharmacokinetic, efficacy and safety data, and case studies of clinical experience of one such concentrate, Biostate. These data are discussed in the context of various therapeutic applications and compared with other factor concentrate products. Data are presented from data on file from the manufacturer; product information and published experimental and clinical pharmacokinetic, safety and efficacy study data; and example case studies of clinical experience.The data discussed herein demonstrate that Biostate has well-established efficacy profiles in the treatment of patients with VWD or HA, with the control of bleeding rated as ‘excellent’, ‘good’ or ‘moderate’ in >90% of patients. In an immune-tolerance induction setting, 73% of patients achieved a complete response following treatment with Biostate. Biostate was generally well tolerated in patients with HA or VWD, with infrequent minor adverse events reported and no reported cases of clinically relevant thrombosis.

<p>Pharmacokinetics, Efficacy and Safety of a Plasma-Derived VWF/FVIII Concentrate (Formulation V) in Pediatric Patients with von Willebrand Disease (SWIFTLY-VWD Study)</p>

“…10 Compared with Haemate P (referred to here as Formulation HP), a VWF/FVIII concentrate also manufactured by CSL Behring that has been available since 1981, Formulation V has a similar VWF:RCo to FVIII:C ratio 11 and multimeric profile. 12 However, Formulation V has a higher concentration and lower volume, 11 and different purification steps during manufacture (ie, heparin/glycine precipitation and gel chromatography compared with multiple precipitation for Formulation HP). 4 The manufacture of Formulation V includes two dedicated viral inactivation steps, namely a solvent detergent treatment at an intermediate stage of the process and dry heat treatment of the freeze-dried product in the final container.…”

The Fate of Von Willebrand Factor Concentrate During Cardiopulmonary Bypass: A Case Report

“…When desmopressin is used as prophylactic treatment for minor surgeries and in bleeding, target VWF: RCo levels of at least 30 IU/dL and preferably >50 IU/dL are recommended [6]. Desmopressin is ineffective for the treatment of qualitative defects found in patients with VWD type 2 as well as the complete absence of VWF in patients with VWD type 3 [4,7,8]. A limitation of this therapy is the diminished response to repeated doses due to depletion of stored VWF [6].…”

“…FVIII/VWF-complex replacement therapy is the definitive treatment for VWD and is the focus of this case review. HPD-FVIII/ VWF concentrates are processed from human plasma to inactivate viruses and prions and were developed as a safer alternative to cryoprecipitate [8]. Guidelines suggest using factor concentrate replacement therapy for significant bleeding or major surgery, or when the patient is refractory to other treatments.…”

“…Guidelines suggest using factor concentrate replacement therapy for significant bleeding or major surgery, or when the patient is refractory to other treatments. HPD-FVIII/ VWF complex concentrates are available for intravenous administration to increase levels of VWF perioperatively [8]. It is recommended to achieve VWF: RCo levels of at least 100 IU/dL is to achieve optimal perioperative coagulation for major surgery [6].…”