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@article{El:2022:10.1111/hae.14550,
author = {El, Alayli A and Petersen, RB and Husainat, NM and Kalot, MA and Aljabiri, Y and Turkmani, H and Britt, A and El-Khechen, H and Shahid, S and Roller, J and Motaghi, S and Mansour, R and Tosetto, A and Abdul-Kadir, R and Laffan, M and Weyand, A and Leebeek, FWG and Arapshian, A and Kouides, P and James, P and Connell, NT and Flood, VH and Mustafa, RA},
doi = {10.1111/hae.14550},
journal = {Haemophilia},
pages = {373--387},
title = {Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review},
url = {http://dx.doi.org/10.1111/hae.14550},
volume = {28},
year = {2022}
}

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TY  - JOUR
AB  - BackgroundVon 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.AimSystematically summarize the evidence on the clinical outcomes of secondary long-term prophylaxis in patients with VWD and severe recurrent bleedings.MethodsWe 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.ResultsWe 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).ConclusionVWD 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.
AU  - El,Alayli A
AU  - Petersen,RB
AU  - Husainat,NM
AU  - Kalot,MA
AU  - Aljabiri,Y
AU  - Turkmani,H
AU  - Britt,A
AU  - El-Khechen,H
AU  - Shahid,S
AU  - Roller,J
AU  - Motaghi,S
AU  - Mansour,R
AU  - Tosetto,A
AU  - Abdul-Kadir,R
AU  - Laffan,M
AU  - Weyand,A
AU  - Leebeek,FWG
AU  - Arapshian,A
AU  - Kouides,P
AU  - James,P
AU  - Connell,NT
AU  - Flood,VH
AU  - Mustafa,RA
DO  - 10.1111/hae.14550
EP  - 387
PY  - 2022///
SN  - 1351-8216
SP  - 373
TI  - Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review
T2  - Haemophilia
UR  - http://dx.doi.org/10.1111/hae.14550
UR  - http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000773328700001&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
UR  - https://onlinelibrary.wiley.com/doi/10.1111/hae.14550
UR  - http://hdl.handle.net/10044/1/96504
VL  - 28
ER  -

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