Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review

Outcomes and Implementation Research Unit, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA. Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada. Department of Internal Medicine, St. Mary's Hospital, Saint Louis, Missouri, USA. The State University of New York at Buffalo Department of Internal Medicine, Buffalo, New York, USA. Lincoln Medical and Mental Health Center, The Bronx, New York, USA. Department of Cardiovascular Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA. Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA. Department of Pediatrics, McMaster University, Hamilton, Ontario, Canada. Department of Hematology/Oncology, University of Kansas Medical Center, Kansas City, Kansas, USA. Hemophilia and Thrombosis Center, Hematology Department, S. Bortolo Hospital, Vicenza, Italy. The Royal Free NHS Foundation Hospital and Institute for Women's Health, University College London, London, UK. Centre for Haematology, Imperial College London, London, UK. Division of Pediatric Hematology Oncology, Department of Pediatrics, University of Michigan Medical School, Ann Arbor, Michigan, USA. Department of Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands. Middle Village, New York, USA. Mary M. Gooley Hemophilia Treatment Center, University of Rochester, Rochester, New York, USA. Department of Medicine, Queen's University, Kingston, Ontario, Canada. Hematology Division, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA. Department of Pediatrics, Medical College of Wisconsin, Versiti Blood Research Institute, Milwaukee, Wisconsin, USA. Division of Nephrology and Hypertension, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas, USA.

Haemophilia : the official journal of the World Federation of Hemophilia. 2022
Abstract
BACKGROUND Von Willebrand Disease (VWD) is a common inherited bleeding disorder. Patients with VWD suffering from severe bleeding may benefit from the use of secondary long-term prophylaxis. AIM: Systematically summarize the evidence on the clinical outcomes of secondary long-term prophylaxis in patients with VWD and severe recurrent bleedings. METHODS We searched Medline and EMBASE through October 2019 for relevant randomized clinical trials (RCTs) and comparative observational studies (OS) assessing the effects of secondary long-term prophylaxis in patients with VWD. We used Cochrane Risk of Bias (RoB) tool and the RoB for Non-Randomized Studies of interventions (ROBINS-I) tool to assess the quality of the included studies. We conducted random-effects meta-analyses and assessed the certainty of the evidence using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. RESULTS We included 12 studies. Evidence from one placebo controlled RCT suggested that VWD prophylaxis as compared to no prophylaxis reduced the rate of bleeding episodes (Rate ratio [RR], .24; 95% confidence interval [CI], .17-.35; low certainty evidence), and of epistaxis (RR, .38; 95%CI, .21-.67; moderate certainty evidence), and may increase serious adverse events RR 2.73 (95%CI .12-59.57; low certainty). Evidence from four before-and-after studies in which researchers reported comparative data suggested that VWD prophylaxis reduced the rate of bleeding (RR .34; 95%CI, .25-.46; very low certainty evidence). CONCLUSION VWD prophylaxis treatment seems to reduce the risk of spontaneous bleeding, epistaxis, and hospitalizations. More RCTs should be conducted to increase the certainty in these benefits.
Study details
Study Design : Systematic Review
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine