Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII‐A deficiency: international phase 3b trial results
Journal of Thrombosis and Haemostasis. 15:1601-1606
ISSN:
1538-7836
DOI:
10.1111/jth.13748
Popis:
SummaryBackground Factor XIII deficiency is a rare, severe congenital bleeding disorder. Monthly prophylaxis with recombinant factor XIII (rFXIII) has demonstrated favorable safety and efficacy in patients ≥6 years and may similarly benefit younger children. Objective To evaluate the long-term safety and efficacy of rFXIII in children