Author(s): Mathias M, Khair K, Hann I, Liesner R
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Abstract We report the use of rituximab (Genentech, San Francisco, CA, USA) in two children with severe haemophilia with inhibitors to factors VIII and IX, which failed to respond to conventional immune tolerance therapy. The treatment was well tolerated by both children. The child with haemophilia B had no clinical improvement or fall in CD19 and he is currently being treated with recombinant activated factor VII (NovoSeven, Novo Nordisk, Denmark) for bleeding episodes. The child with haemophilia A had a good clinical response with a negative inhibitor assay at 11 months follow-up.
This article was published in Br J Haematol
and referenced in Journal of Genetic Syndromes & Gene Therapy