Vonicog alfa (recombinant von Willebrand factor) Outcomes UK
Research type
Research Study
Full title
Vonicog alfa (recombinant von Willebrand factor) Treatment Outcomes in von Willebrand Disease in the UK: a Retrospective Chart Review Study.
IRAS ID
291991
Contact name
Michael Laffan
Contact email
Sponsor organisation
Takeda Pharmaceuticals
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 8 months, 14 days
Research summary
This retrospective chart review study will collect data on real world use of vonicog alfa (rVWF) within its licensed indication in the UK, in adult patients (aged 18 years or older at the time of first treatment with rVWF) with congenital von Willebrand disease (VWD) to treat spontaneous and traumatic bleeds and in the prevention and management of surgical bleeds including post-operative bleeding.
Summary of results: A Study to Learn About the Effects of Recombinant Von Willebrand Factor in Adults With Von Willebrand Disease in the United Kingdom
Von Willebrand Disease (VWD) is a condition that causes excessive bleeding due to problems with blood clotting. The main goal of the study was to observe the effect of the recombinant Von Willebrand Factor (rVWF) treatments among adults with inherited VWD in the United Kingdom (UK) in 7 hospitals in the UK.
To do this, researchers looked at participants who had been given rVWF to treat sudden, unexpected bleeding and to prevent or treat bleeding connected to surgery. Researchers looked at participants’ data who first received rVWF treatment between 1 October 2020 and 30 June 2022. They checked their medical records to gather information about their bleeding, treatments, hospitalizations, and surgeries at the time of their first use of rVWF, as well as in the 12 months before and 3-12 months afterwards.
A total of 34 participants were included in the study. Of these, 32 were not receiving long-term preventative treatment at the time they were first given rVWF. The results below describe the first use of rVWF (referred to as the index period) in these 32 participants. The average age in these participants was 48 years and 22/32 participants were female.
Overall, 12 participants were treated with rVWF for bleeds during the index period. Most bleeding episodes were mild in severity. rVWF was successful in resolving all of these bleeds, as indicated by the treating clinician.
A total of 20 participants were treated with rVWF to prevent or treat surgical bleeds during the index period. 12 participants had major surgery, including 1 related to an injury, and 8 participants had minor surgery. In total, rVWF was given to 11 participants before surgery, 9 participants during surgery, and 12 participants after surgery (participants may have received rVWF at multiple timepoints). rVWF was successful in resolving any bleeding that occurred in these participants at index, as indicated by the treating clinician.
Researchers collected information on participants’ bleeding episodes and surgeries in the 3 to 12 months after the index event. They checked if these were managed with rVWF treatment. rVWF treatment was used to manage 12/13 bleeds and 10/13 surgeries that occurred during this period. In addition, they collected information on bleeding episodes and surgeries that occurred in the 12 months before first use of rVWF. A total of 5 bleeds and 4 surgeries were reported for this period.
3 participants had complications due to surgical bleeds. This included blood clots in veins, inability to pass urine due to blood clots, and high blood pressure, which were all deemed either ‘not related’ or ‘unlikely to be related’ to the rVWF treatment.
This study showed that overall, the effects of rVWF treatments were consistent with those seen in previous clinical studies. The results showed that rVWF could help participants with VWD in the UK when used during sudden bleeding events or for the prevention and treatment for bleeding during surgeries.This summary gives the main results of this single study. Other studies may give different results. Researchers look at the results of many studies to decide which medicines work best and are safest for patients.
More information about the study is available at:
https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Cleedswest.rec%40hra.nhs.uk%7Ceb2c586eac3043f1f22e08dd19d5998e%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638695127385474790%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=ahU62tNiGJrO4v19lQFEuzgJr8PfGHRP0FgG2Tw1hGM%3D&reserved=0 (search this website using the study number TAK-577-5001)
https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fwww.clinicaltrials.takeda.com%252F%2FNBTI%2FR-K5AQ%2FAQ%2Fa497a2bf-49d4-4e4b-955d-074813b4c21a%2F1%2FCzTvV3lHQH&data=05%7C02%7Cleedswest.rec%40hra.nhs.uk%7Ceb2c586eac3043f1f22e08dd19d5998e%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638695127385495535%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=hO7DuWcOZInHRj1tz1HvCUsY4YqtytjTeX5QHkgakHo%3D&reserved=0 (use the detailed search using the Study Identifier TAK-577-5001)Thank you to all of the participants who took part in this study. The study was sponsored by Takeda UK.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
21/YH/0009
Date of REC Opinion
26 Feb 2021
REC opinion
Further Information Favourable Opinion