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ASH 2025 | Plasma-derived VWF/FVIII prophylaxis in children under 6 with VWD: first results from WIL-33
Akshat Jain, MD, MPH, FRCP, Loma Linda University, San Bernardino, CA, provides insight into the WIL-33 trial (NCT04953884), an international study that investigated the use of prophylaxis with plasma-derived von Willebrand factor (VWF)/factor VIII (FVIII) concentrate in children under 6 with von Willebrand disease (VWD). Dr Jain highlights that the study, which he led over the past six years, has provided the first demonstration of the safety and efficacy of IV VWF replacement therapy for controlling severe bleeding episodes in young children, thereby guiding clinical practice and advancing the treatment of this severe lifelong bleeding disorder. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
Again, very proud of WIL-33, which is an international multi-country study that I have led over the past six years, including Asia, Africa, Middle East, United States, Canada, and other parts of the world, to target treatment for, again, another marginalized group of bleeding disorder patients which is children under the age of six years. So pediatric patients with severe symptomatic von Willebrand disease, who suffer from bleeding right from the time that they are born but there is no uniform understanding diagnostic platform and or treatment paradigm that has been set out to treat such young children with the life-threatening bleeding disorder...
Again, very proud of WIL-33, which is an international multi-country study that I have led over the past six years, including Asia, Africa, Middle East, United States, Canada, and other parts of the world, to target treatment for, again, another marginalized group of bleeding disorder patients which is children under the age of six years. So pediatric patients with severe symptomatic von Willebrand disease, who suffer from bleeding right from the time that they are born but there is no uniform understanding diagnostic platform and or treatment paradigm that has been set out to treat such young children with the life-threatening bleeding disorder. So we set out about five years ago starting the WIL-33 study, conceptualizing to once and for all create a paradigm by testing pharmacokinetics and pharmacodynamics of IV von Willebrand factor replacement therapy, the WIL-8 molecule, and seeing how well it works in children so young, which has never ever been done in the past before at such a large level in a scientific manner.
Fast forward five years later at completion of study we’ve gotten an amazing set of data which is for the first time demonstrating the safety and efficacy of using IV von Willebrand factor replacement to control severe bleeding episodes that are predominant, prevalent and affect the quality of life of all these children significantly. Helping clinicians now know how to treat children under six years of age in a very organic and a scientific manner in a stepwise approach to decide how much factor, how frequently, in what subgroup of patients can be given thereby now guiding clinical practice. A study that has been done despite two major wars and a pandemic, we are very proud that we were successfully able to recruit subjects and we have really thanked our investigators all around the world that I had the pleasure of leading and able to collect and be able to present that data at such an august podium at the American Society of Hematology meeting has really helped us add to the literature and advanced science in how we treat children with von Willebrand disease which is a severe lifelong bleeding disorder.
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