UK researchers say that effective prophylaxis and treatment for children with congenital thrombocytopenic purpura (TTP) is possible using a US-sourced plasma-derived factor VIII concentrate called BPL 8Y.
The team, led by Ri Liesner, from Great Ormond Street Hospital for Children in London, makes this recommendation based on their study in which they recorded the outcome of BPL 8Y treatment for seven children with TTP.
There are many conventional methods of plasma infusion for TTP, characterized by a deficiency in ADAMTS-13, but these can entail fluid overload, allergic reactions, or the risk of transfusion-transmitted infections, the scientists explain.
They add that BPL 8Y offers a viable alternative to conventional methods as it is a virally inactivated plasma infusion containing ADAMTS-13 that is not thought to confer the same side effects, they explain.
Writing in the British Journal of Haematology, the authors state that six of the children had a history of severe neonatal unconjugated hyperbilirubinemia and thrombocytopenia, whereas the seventh child did not but had suffered three episodes of acute TTP.
All of the children presented with ADAMTS-13 activity of less than 5%, with a median platelet count at presentation of 31 x 109/1.
The patients received 15-30 iu/kg BPL 8Y, and from nadir platelet counts at presentation ranging from 5 x 109/1 to 120 x 1091, the researchers observed platelet counts rise to between 45 x 109/1 and 120 x 109/l an average of 48 hours after BPL 8Y treatment.
Liesner et al conclude: "Treatment with virally treated fresh frozen plasma is efficacious, but BPL 8Y, and intermediate-purity US-sourced plasma, allows smaller volumes to be given both acutely and prophylactically, as home treatment."
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