The role of primary prophylactic factor replacement therapy in children with severe factor X deficiency.

Abstract

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.

• Authors: Corrina McMahon, Judith Smith, Collette Goonan, Mary Byrne, Owen P Smith
• Journal: British journal of haematology (Br J Haematol) Vol. 119 Issue 3 Pg. 789-91 (Dec 2002) ISSN: 0007-1048 [Print] England
• PMID: 12437661 (Publication Type: Case Reports, Journal Article)

Chemical References

• Blood Coagulation Factors
• prothrombin complex concentrates

Topics

• Adolescent
• Blood Coagulation Factors (therapeutic use)
• Catheters, Indwelling
• Child
• Factor X Deficiency (genetics, prevention & control)
• Female
• Hemorrhagic Disorders (etiology, prevention & control)
• Humans
• Male
• Pedigree