Characterization and management of patients with hereditary factor X deficiency: A case series.

ABSTRACT:

Background

Hereditary factor X deficiency (HFXD) is an ultra-rare, autosomal recessive bleeding disorder that results in reduced factor X coagulant activity (FX:C). HFXD is traditionally classified by severity as severe (FX:C <1%), moderate (FX:C = 1%-5%), or mild (FX:C = 6%-10%). Treatment aims to replace the deficient factors, but there are no standardized guidelines for management.

Study design and methods

This multicenter retrospective chart review examined the diagnostic features, bleeding patterns, and current management strategies for HFXD patients in the United States. Patients were classified based on the revised European Network of Rare Bleeding Disorders (EN-RBD) criteria: severe (FX:C <10%), moderate (FX:C = 10%-40%), and mild (FX:C >40%). Data, including demogra

SUBMITTER: Chitlur M 

PROVIDER: S-EPMC12531909 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature