1. Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review
- Author
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Abdallah El Alayli, Romina Brignardello Petersen, Nedaa M. Husainat, Mohamad A. Kalot, Yazan Aljabiri, Hani Turkmani, Alec Britt, Hussein El‐Khechen, Shaneela Shahid, John Roller, Shahrzad Motaghi, Razan Mansour, Alberto Tosetto, Rezan Abdul‐Kadir, Michael Laffan, Angela Weyand, Frank W.G. Leebeek, Alice Arapshian, Peter Kouides, Paula James, Nathan T. Connell, Veronica H. Flood, and Reem A. Mustafa
- Subjects
bleeding disorder ,Science & Technology ,epistaxis ,bleeding episodes ,FACTOR CONCENTRATE ,1103 Clinical Sciences ,General Medicine ,Hematology ,EFFICACY ,Hospitalization ,von Willebrand Diseases ,Cardiovascular System & Hematology ,QUALITY-OF-LIFE ,WILATE ,SAFETY ,Chronic Disease ,von Willebrand Factor ,MODERATE ,Humans ,COHORT ,prophylaxis ,Hemophilia ,Life Sciences & Biomedicine ,Genetics (clinical) ,Von Willebrand Disease - 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.
- Published
- 2022