Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy
Congenital factor V deficiency is an inherited bleeding disorder due to reduced plasma levels of factor V (FV) and characterized by mild to severe bleeding symptoms.
Features include common findings: Epistaxis and Joint hemorrhage; and sometimes findings: Menorrhagia, Bruising susceptibility, Gingival bleeding, and Blood in the urine (hematuria) and others. 25 total HPO annotations.
Organ System | Phenotype Count | Example Features |
|---|---|---|
Blood and immune system | 10 | Abnormal bleeding tendency (abnormal bleeding), Reduced coagulation factor V activity, Prolonged bleeding time |
Bones and joints | 1 | Joint hemorrhage |
Kidneys and urinary system | 1 | Blood in the urine (hematuria) |
Digestive system | 1 | Gastrointestinal hemorrhage |
Heart and blood vessels | 1 | Intracranial hemorrhage |
Venous thromboembolism (VTE) is the primary clinical manifestation of factor V Leiden thrombophilia . The most common site for VTE is the legs, but upper-extremity, cerebral, and superficial venous thrombosis may also occur. The relative risk for VTE is increased approximately three- to eightfold in factor V Leiden variant heterozygotes . Lower relative risks (four- to fivefold) were reported in two large meta-analyses . Despite the increase in relative risk, the overall annual incidence of a first VTE is low in heterozygotes, approximately 0.5% . The reported adjusted hazard ratio (HR) for VTE in heterozygotes compared with controls was 2.7 (95% confidence interval [CI] 1.8-3.8) .
Source: GeneReviews — "Factor V Leiden Thrombophilia"
F5 encodes coagulation factor V (2,224 aa). Central regulator of hemostasis. It serves as a critical cofactor for the prothrombinase activity of factor Xa that results in the activation of prothrombin to thrombin Highest expression in Liver (34.2 TPM) and Whole Blood (19.2 TPM).
Congenital factor V deficiency is caused by mutations in the F5 gene on chromosome 1.
The F5 protein participates in factor V activation peptide, factor V (738-1046), and F5 R334T, F5 I387T pathways.
F5 is classified as a druggable target (Druggable Genome and Enzyme categories) with score 4.5.
Factor V Leiden thrombophilia should be suspected in individuals with the following clinical, laboratory, and family history findings:
Clinical findings. A history of one or recurrent venous thromboembolism (VTE) manifesting as deep vein thrombosis (DVT) or pulmonary embolism (PE), especially at a young age and in the absence of strong risk factors for VTE
Laboratory findings. Low activated protein C (APC) resistance on APC resistance qualitative and quantitative assays
Source: GeneReviews — "Factor V Leiden Thrombophilia"
The differential diagnosis of venous thromboembolism (VTE) includes several other inherited thrombophilic disorders, including those caused by other variants in F5, and acquired thrombophilic disorders (outside of the scope of this GeneReview). Prothrombin thrombophilia is characterized by VTE manifesting most commonly in adults as deep vein thrombosis (DVT) in the legs or pulmonary embolism. The clinical expression of prothrombin thrombophilia is variable; many individuals heterozygous or homozygous for the 20210GA F2 variant never develop thrombosis, and while most heterozygotes who develop thrombotic complications remain asymptomatic until adulthood, some have recurrent thromboembolism before age 30 years.
Source: GeneReviews — "Factor V Leiden Thrombophilia"
Genetic testing for F5 is available. Testing is considered confirmatory for diagnosis.
No approved treatments are currently available for congenital factor V deficiency. The disease remains an area of unmet medical need.
To assess the risk for venous thromboembolism (VTE) in an individual found to have a factor V Leiden variant, the following are recommended:
DNA analysis for prothrombin thrombophilia (F2 variant c.*97GA, commonly known as 20210GA)
Multiple phospholipid-dependent coagulation assays for a lupus inhibitor
Serologic assays for anticardiolipin antibodies and anti-beta-2-glycoprotein 1 antibodies
For high-risk individuals (i.e., those with a history of recurrent VTE, especially at a young age, or those with strong family history of VTE at a young age), evaluation should also include assays of:
Protein C activity
Antithrombin activity
Protein S activity or free protein S antigen
Note: Measurement of the following is NOT recommended:
Plasma concentration of homocysteine, as no data support a change in duration of anticoagulation or the use of vitamin supplementation in individuals with hyperhomocysteinemia and a history of VTE
MTHFR variants, as no clinical rationale for this testing exists
Factor VIII and other clotting factor levels
Treatment of Manifestations
The management of individuals with factor V Leiden thrombophilia depends on the clinical circumstances. The first acute thrombosis should be treated according to current guidelines . For initial treatment of VTE, current guidelines suggest a direct oral anticoagulant (dabigatran, edoxaban, rivaroxaban, or apixaban) over warfarin because of a lower bleeding risk and gr...
Source: GeneReviews — "Factor V Leiden Thrombophilia"
View trials for congenital factor V deficiency
Individuals receiving long-term anticoagulation require periodic reevaluation of their clinical course to confirm that the benefits of anticoagulation continue to outweigh the risk of bleeding. Factor V Leiden heterozygotes who do not require long-term anticoagulation may benefit from evaluation prior to exposure to circumstantial risk factors such as surgery or pregnancy.
Source: GeneReviews — "Factor V Leiden Thrombophilia"
Phenotype severity distribution: 2 common features.
Estimated prevalence: 1-9 in 1,000,000 (Rare).
No clinical trials have been registered for congenital factor V deficiency.
9 publications have been identified in PubMed for congenital factor V deficiency. Research spans Case Report / Case Series (44%), Epidemiology / Natural History (33%), and Review / Meta-Analysis (22%).
Stoj KM (2026). [PMID: 35593819](https://pubmed.ncbi.nlm.nih.gov/35593819/). *Unknown Journal*. [Review / Meta-Analysis]
Pshenichnikova O (2026). [PMID: 42074284](https://pubmed.ncbi.nlm.nih.gov/42074284/). *Int J Mol Sci*. [Epidemiology / Natural History]
Sava C (2025). [PMID: 39740908](https://pubmed.ncbi.nlm.nih.gov/39740908/). *In vivo (Athens, Greece)*. [Epidemiology / Natural History]
Franchini M (2025). [PMID: 39191405](https://pubmed.ncbi.nlm.nih.gov/39191405/). *Seminars in thrombosis and hemostasis*. [Review / Meta-Analysis]
Guilbault C (2025). [PMID: 40788998](https://pubmed.ncbi.nlm.nih.gov/40788998/). *Journal of pediatric hematology/oncology*. [Case Report / Case Series]
Yang Y (2025). [PMID: 40421928](https://pubmed.ncbi.nlm.nih.gov/40421928/). *Haemophilia : the official journal of the World Federation of Hemophilia*. [Epidemiology / Natural History]
Bendarkawi Y (2025). [PMID: 40296097](https://pubmed.ncbi.nlm.nih.gov/40296097/). *Journal of medical case reports*. [Case Report / Case Series]
Zhang H (2025). [PMID: 40684283](https://pubmed.ncbi.nlm.nih.gov/40684283/). *Hematology (Amsterdam, Netherlands)*. [Case Report / Case Series]
Zhang Y (2024). [PMID: 39560563](https://pubmed.ncbi.nlm.nih.gov/39560563/). *Medicine*. [Case Report / Case Series]
Data assembled from 8 of 12 sources · Last updated Sep 19, 2026, 1:00 PM UTC
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
AI-curated news mentioning congenital factor V deficiency
Updated Mar 2, 2026
A recent publication discusses the current management and care gaps for Factor II and Factor V deficiencies, highlighting the need for therapeutic advancements. The article calls for increased research and innovation to improve patient outcomes in these rare bleeding disorders.