Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A
Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A
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.
796-804
Batty, Paul
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Austin, Steve K.
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Khair, Kate
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Millar, Carolyn M.
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Palmer, Ben
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Rangarajan, Savita
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Stümpel, Jan-Phillip
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Thanigaikumar, Murugaiyan
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Yee, Thynn T.
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Hart, Daniel P.
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1 March 2017
Batty, Paul
965f536b-8873-466e-9c5f-bf8cb80f6591
Austin, Steve K.
b5478f39-59da-4945-a0d1-c387a5cfedea
Khair, Kate
8eed2e8f-c79b-4172-be6e-8755bf39e4cc
Millar, Carolyn M.
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Palmer, Ben
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Rangarajan, Savita
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Stümpel, Jan-Phillip
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Thanigaikumar, Murugaiyan
ea4575a5-b9f3-43cb-9ee3-14b4def1df47
Yee, Thynn T.
a1e7ff43-fd54-4cf8-bded-78126fc587f4
Hart, Daniel P.
806f3603-67f9-4f07-a8b7-9b183eb55c98
Batty, Paul, Austin, Steve K., Khair, Kate, Millar, Carolyn M., Palmer, Ben, Rangarajan, Savita, Stümpel, Jan-Phillip, Thanigaikumar, Murugaiyan, Yee, Thynn T. and Hart, Daniel P.
(2017)
Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A.
British Journal of Haematology, 176 (5), .
(doi:10.1111/bjh.14543).
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.
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Accepted/In Press date: 3 November 2016
e-pub ahead of print date: 15 February 2017
Published date: 1 March 2017
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Local EPrints ID: 442856
URI: http://eprints.soton.ac.uk/id/eprint/442856
ISSN: 0007-1048
PURE UUID: 7b5752ef-7efd-4712-81fc-78baeb453bdd
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Date deposited: 29 Jul 2020 16:34
Last modified: 17 Mar 2024 04:02
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Contributors
Author:
Paul Batty
Author:
Steve K. Austin
Author:
Kate Khair
Author:
Carolyn M. Millar
Author:
Ben Palmer
Author:
Jan-Phillip Stümpel
Author:
Murugaiyan Thanigaikumar
Author:
Thynn T. Yee
Author:
Daniel P. Hart
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