rVWF Safely, Effectively Manages Perioperative Bleeding in Pediatric Patients: Shayla Bergmann, MD

Recombinant von Willebrand factor (rVWF) could reshape how children with bleeding disorders are managed during surgery, says Shayla Bergmann, MD, a clinical professor in pediatric hematology at the Medical University of South Carolina. In a landmark multicenter study presented at the American Society of Hematology (ASH) 2025 meeting, rVWF was both highly effective and remarkably safe—showing no adverse events—and offered a more convenient option than traditional plasma-derived therapies among pediatric patients with von Willebrand disease (VWD).

This transcript was lightly edited; captions were auto-generated.

Transcript

Can you provide a brief overview of the study presented at ASH 2025?

Our recombinant von Willebrand factor study involving the perioperative management of bleeding in pediatric patients with severe bundle of brain disease has some data that are interim results from their phase 3 multicenter study, as well as their phase 3b, which is the continuation study in pediatrics. The goal was to look at the safety and efficacy of recombinant von Willebrand factor, or Vonvendi, in the perioperative setting in pediatric patients, so those are patients less than 18 years of age, and to evaluate not only the safety and efficacy, but the secondary end point, [which] would be the amount of bleeding that may have occurred.

How effective was rVWF in preventing or controlling perioperative bleeding in pediatric patients with VWD, and did effectiveness differ by VWD type or severity?

To give you a little bit of background about the study design, it was an open-label, prospective study that was conducted in 45 sites, both in the US and Europe. It pulled patients from our pediatric on-demand trial as well as our individual surgery study and then also has patients from the extension part of those trials. There are different parts that you could qualify if you needed elective surgery or if you had emergent surgery, and the bleeding control was evaluated not only by the primary investigator, or the PI, but also by the surgeon that was involved in the procedure, both intraoperatively and then post op. There was a 4-scale rating to help grade the efficacy and the amount of bleeding that occurred, and that was based on an excellent, good, fair, or poor scale, so a 4-point scale. All procedures that involved the use of recombinant von Willebrand factor were all graded as excellent, not only intraoperatively but also postoperatively, not only by the surgeons but also by the PI, so 100%.