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The role of primary prophylactic factor replacement therapy in children with severe factor X deficiency
Author(s) -
McMahon Corrina,
Smith Judith,
Goonan Collette,
Byrne Mary,
Smith Owen P.
Publication year - 2002
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.2002.03932.x
Subject(s) - medicine , bleed , umbilical cord , coagulation , surgery , factor ix , pediatrics , incidence (geometry) , clotting factor , immunology , physics , optics
Summary. Severe factor X (FX) deficiency is one of the severest inherited coagulation disorders. Clinical manifestations include umbilical cord, mucosal, joint and central nervous system bleeding. Four Irish children with severe FX deficiency presented with umbilical cord bleeding. One developed an intraperitoneal haemorrhage and another an intracranial bleed. Prophylaxis, using intermediate purity Factor IX concentrate, was commenced within the first month of life, necessitating the insertion of central venous access devices in two of the children. All children have normal joint function, suggesting that prophylaxis commenced early in life reduces the incidence of arthropathy and improves quality of life.