Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A
File(s)BJH - Non Severe Haemophilia Audit Accepted.docx (1.6 MB)
Accepted version
Author(s)
Type
Journal Article
Abstract
Inhibitor formation in non-severe haemophilia A is a life-long risk and associated with morbidity and mortality. There is a paucity of data to understand real-world inhibitor screening practice. We evaluated the treatment burden, haemostatic strategies, F8 genotyping and inhibitor screening practices in non-severe haemophilia A in seven London haemophilia centres. In the 2-year study period, 44% (377/853) patients received at least one haemostatic treatment. Seventy-nine percent of those treated (296/377) received factor VIII (FVIII) concentrate. F8 genotype was known in 88% (331/377) of individuals. Eighteen per cent (58/331) had ‘high-risk’ F8 genotypes. In patients with ‘standard-risk’ F8 genotypes treated on-demand with FVIII concentrate, 51·3% episodes (243/474) were screened within 1 year. However, poor screening compliance was observed after ‘high-risk’ treatment episodes. In patients with ‘standard-risk’ F8 genotypes, 12·3% (28/227) of treatment episodes were screened in the subsequent 6 weeks after surgery or a bleed requiring ≥5 exposure days. Similarly, in the context of ‘high-risk’ F8 genotypes after any FVIII exposure, only 13·6% (12/88) of episodes were screened within 6 weeks. Further study is required to assess optimal practice of inhibitor screening in non-severe haemophilia A to inform subsequent clinical decisions and provide more robust prevalence data to further understand the underlying immunological mechanism.
Date Issued
2017-02-15
Date Acceptance
2016-11-03
Citation
British Journal of Haematology, 2017, 176 (5), pp.796-804
ISSN
1365-2141
Publisher
Wiley
Start Page
796
End Page
804
Journal / Book Title
British Journal of Haematology
Volume
176
Issue
5
Copyright Statement
© 2017 John Wiley & Sons Ltd. This is the accepted version of the following article, which has been published in final form at http://onlinelibrary.wiley.com/doi/10.1111/bjh.14543/abstract
Identifier
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000395191500013&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
Subjects
Science & Technology
Life Sciences & Biomedicine
Hematology
haemophilia
non-severe
inhibitor
screening
audit
PREVIOUSLY UNTREATED PATIENTS
PREANALYTICAL HEAT-TREATMENT
VIII ANTIBODY DETECTION
FACTOR-IX
MISSENSE MUTATIONS
BETHESDA ASSAY
RISK-FACTORS
ELISA
DIAGNOSIS
COHORT
Publication Status
Published