Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A.

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Batty, P; Austin, SK; Khair, K; Millar, CM; Palmer, B; Rangarajan, S; Stümpel, J-P; Thanigaikumar, M; Yee, TT; Hart, DP (2017) Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A. Br J Haematol, 176 (5). pp. 796-804. ISSN 1365-2141 https://doi.org/10.1111/bjh.14543
SGUL Authors: Austin, Steve

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Official URL: https://doi.org/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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This is the peer reviewed version of the following article: Batty, P., Austin, S.K., Khair, K., Millar, C.M., Palmer, B., Rangarajan, S., Stümpel, J.-P., Thanigaikumar, M., Yee, T.T. and Hart, D.P. (2017), Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A. Br J Haematol, 176: 796-804, which has been published in final form at https://doi.org/10.1111/bjh.14543. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions. This article may not be enhanced, enriched or otherwise transformed into a derivative work, without express permission from Wiley or by statutory rights under applicable legislation. Copyright notices must not be removed, obscured or modified. The article must be linked to Wiley’s version of record on Wiley Online Library and any embedding, framing or otherwise making available the article or pages thereof by third parties from platforms, services and websites other than Wiley Online Library must be prohibited.

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