Congenital Factor VII Deficiency: A Case Study of Four Family Members

先天性凝血因子VII缺乏症:四名家庭成员的病例研究

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Abstract

Congenital factor VII (FVII) deficiency is a rare genetic disorder with autosomal recessive inheritance, characterized by molecular and clinical heterogeneity. This article reports four Moroccan cases of FVII deficiency within the same family, two of which were associated with Gilbert's syndrome. The index case was a 15-year-old girl with a history of menorrhagia and jaundice. Upon examination, she presented with indirect hyperbilirubinemia, iron deficiency anemia, and a prolonged prothrombin time (PT). FVII deficiency was confirmed with a factor VII level of 45%. Her mother, a 30-year-old woman with sarcoidosis and a history of postpartum hemorrhage, was found to have jaundice and an FVII deficiency at 42%. The brother of the index case, an eight-year-old boy who was asymptomatic, was discovered to have FVII deficiency during family screening, with a level of 41%. Similarly, the six-year-old sister, also asymptomatic, had an FVII level of 33%. The prevalence of homozygous FVII deficiency is rare, and the risk is increased in consanguineous marriages. Clinical presentations vary widely, ranging from asymptomatic cases to severe bleeding episodes. Diagnosis is based on a prolonged PT with a normal activated partial thromboplastin time (aPTT) and is confirmed through FVII assays. Severe cases may require prophylactic treatment, and recombinant activated FVII (rFVIIa) is the recommended therapy for bleeding episodes. In conclusion, FVII deficiency is the most common of the rare coagulation factor deficiencies. This study explores familial congenital factor VII deficiency, characterized by varied presentations from asymptomatic to mild hemorrhagic symptoms. Early diagnosis and family screening are essential. Management includes symptomatic treatment, tranexamic acid for menorrhagia, and fresh frozen plasma or recombinant factor VIIa for bleeding episodes.

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