Abstract Number: OC 39.4
Meeting: ISTH 2022 Congress
Background: Severe factor VII (FVII) deficiency is a rare bleeding disorder. Bleeding phenotype and management in children with severe FVII deficiency is poorly described.
Aims: This retrospective, observational study aimed to characterise bleeding phenotype and management in individuals aged < 18 years with severe FVII deficiency.
Methods: The UK National Haemophilia Database was scrutinised for individuals aged < 18 years with baseline FVII < 0.05 IU/mL. Individual centres were contacted to provide additional data.
Results: 27 individuals were identified, 16 males and 11 females, from 9 centres. Additional data were available for 26 children, aged 3 months-15 years (median age: 10.5 years). FVII was 10 years), intracranial haemorrhage (ICH, 4), muscle haematoma(2), haemarthrosis(2), cephalohaematoma(1), dental extraction bleeding(1), gastrointestinal bleeding(1), umbilical cord bleeding(1). ICH occurred at birth, day 4 of life, 2 and 6 months of age and none required surgical management. Four individuals had no bleeding symptoms.
Ten receive prophylaxis with FVII concentrate, 9 recombinant and 1 plasma-derived. Prophylaxis was commenced for significant bleeding (4 ICH, 1 muscle haematomata, 1 mucosal bleeding) or for primary prophylaxis (4). Only one commenced prophylaxis after 2 months of age.
Treatment-related complications were reported in two individuals, FVII inhibitor and multiple central venous line-related thromboses.
Conclusion(s): Serious bleeding in severe FVII deficiency, including ICH, occurs early in life. Frequent non-severe bleeding symptoms are mouth/nose bleeding and menorrhagia. More than one third require prophylaxis and this is started early in life.
To cite this abstract in AMA style:Biss T, Motwani J, Grainger J, Mathias M, Stokley S, Kelly A, Payne J, Alamelu J, Green S, Chalmers E, Bhatnagar N. Clinical characteristics and management of children with severe factor VII deficiency- analysis of a United Kingdom cohort [abstract]. https://abstracts.isth.org/abstract/clinical-characteristics-and-management-of-children-with-severe-factor-vii-deficiency-analysis-of-a-united-kingdom-cohort/. Accessed September 27, 2022.
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