Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort

Collins, Peter William ORCID: https://orcid.org/0000-0002-6410-1324, Mathias, M., Hanley, J., Keeling, D., Keenan, R., Laffan, M., Parry, D. and Liesner, R. 2009. Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort. Journal of Thrombosis and Haemostatis 7 (5) , pp. 787-794. 10.1111/j.1538-7836.2009.03332.x

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Abstract

Background and objectives: The management of patients with severe hemophilia A and inhibitors to factor VIII (FVIII) resistant to standard immune tolerance is challenging. There have been recent case reports of the successful use of rituximab in up to 57% of patients as part of rescue immune tolerance regimens. Because case reports and small series are prone to the potential bias of reporting good outcomes and relatively short follow up, a consecutive cohort of all patients treated in the UK with prolonged follow up was analyzed. Methods: A national survey of all Comprehensive Care Haemophilia Cente in the UK. Results: A total of 15 patients were reported of whom six (40%) achieved a negative inhibitor titer by Bethedsa assay. Durable responses were unusual, observed in only 14% of cases. Clinically significant responses with either a negative inhibitor or an inhibitor titer < 5 BU mL)1 and no spontaneous bleeding with FVIII replacement were observed in seven (47%) cases. Concomitant use of FVIII appeared to be important. Of the 12 patients treatedwith rituximab andFVIII, six (50%) achieved a negative inhibitor titer and seven (58%) had a clinically beneficial response. None of the three patients treated without FVIII responded. Conclusions: These data suggest that the use of rituximab combinedwith FVIII is a potentially useful treatment for patients with inhibitors resistant to standard immune tolerance, although sustained inhibitor eradication is uncommon.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Medicine Systems Immunity Research Institute (SIURI)
Subjects:	R Medicine > R Medicine (General)
Uncontrolled Keywords:	hemophilia A; immune tolerance; inhibitor; rituximab
Publisher:	Wiley-Blackwell
ISSN:	1538-7933
Last Modified:	20 Oct 2022 08:09
URI:	https://orca.cardiff.ac.uk/id/eprint/27400

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