Efficacy and safety of a VWF/FVIII concentrate (wilate^®) in inherited von Willebrand disease patients undergoing surgical procedures

Abstract

Introduction Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate® is a plasma‐derived, double virus‐inactivated, highly purified, freeze‐dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1. Aim To investigate the efficacy and safety of wilate® in maintaining haemostasis in VWD patients undergoing surgical procedures. Methods This prospective, open‐label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate®. Twenty‐one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra‐ and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed. Results Surgical prophylaxis with wilate® was successful in 29 of 30 procedures. The overall rate of success was 96.7% (98.75% CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed. Conclusions Wilate® demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.

Fibrinogen concentrate for treatment of bleeding and surgical prophylaxis in congenital fibrinogen deficiency patients

“…The investigator's clinical assessment of hemostatic efficacy for all BEs treated with HFC during the study period was based on a 4‐point objective scale (excellent, good, moderate, and none; Table S1). Previous studies demonstrated the utility of objective criteria for the clinical investigation of other congenital bleeding disorders . All clinical efficacy assessments were adjudicated by an independent data monitoring and endpoint adjudication committee (IDMEAC) according to the same 4‐point objective scale using data collected from the case report forms and information provided by the investigator.…”

Knowledge and Attitudes Towards Clinical Trial Participation in Oman: A cross-sectional study

“…Hussain Al-Lawati, 1 Khalid Al-Baimani, 2 Mahra Al-Zadjali, 3 Nasra Al-Obaidani, 3 Ziyana Al-Kiyumi, 3 A clinical trial is a prospective study of human subjects in which the effect of one or more interventions is compared with a control group in order to answer important clinical questions. 1 It is a major type of investigative research methodology that assesses new interventions in order to provide non-biased insight into their effectiveness, as well as their potential adverse events and complications.…”

“…The results of clinical trials are considered to constitute high-quality evidence in clinical medicine and have contributed to vast improvements in the quality of healthcare provision in various fields. [2][3][4] However, according to the clinical trial registry of the National Library of Medicine (Bethesda, Maryland, USA), only 20 registered clinical trials to date have involved Oman, most of which were sponsored by various pharmaceutical companies. 5 While patients' attitudes towards participating in clinical trials have been analysed in other areas of the world, they have not yet been studied in Oman; as such, the reasons underlying low clinical trial participation among the local population remain unclear.…”

Replacement therapy during surgery in von Willebrand disease needs personalization

“…One product was almost devoid of FVIII with a ratio of 1:≤0.2 (Wilfact/Wilfactin); another (in 2 studies) had a 1:1 ratio (Wilate), and in 2 studies, the same brand was used as in the study reported by Hazendonk et al with a ratio of 1:0.4 (Humate‐P) (all ratios given are approximate). Accumulation of FVIII after repeated dosing was not seen using the 1:1 ratio concentrate or the purified concentrate, whereas the studies using the product with 1:0.4 ratio reported some degree of accumulation after multiple dosing, although not to the same degree as reported in the study by Hazendonk et al. Lethagen and colleagues reported one case of a pulmonary embolism without sequelae in a patient with multiple risk factors.…”

“…All these reasons hamper the ability to report findings from large and well‐defined cohorts of surgical procedures in VWD, although a few such studies have been published for specific concentrates . Altogether, the 5 studies report 257 procedures with excellent or good efficacy ranging from 91% to 100%.…”