Is a 54-year-old female with an ENG (Endoglin) mutation Variant of Uncertain Significance (VUS), no history of epistaxis (nosebleeds), and no arteriovenous malformations (AVMs) but with a 2.9 cm inferior mesenteric venous varix likely to have Hereditary Hemorrhagic Telangiectasia (HHT)?

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Is This Patient Likely to Have HHT?

This patient is unlikely to have HHT based on current evidence, as she meets only 1 of 4 Curaçao criteria (possible visceral lesion) and has a variant of uncertain significance rather than a confirmed pathogenic mutation. 1

Applying the Curaçao Diagnostic Criteria

The diagnosis of HHT requires clinical assessment using the Curaçao criteria, which includes four features: 1, 2

  • Spontaneous and recurrent epistaxis: Absent in this patient 1
  • Multiple telangiectasias at characteristic sites (lips, oral cavity, fingers, nose): Not mentioned, presumably absent 1
  • Visceral lesions (pulmonary AVMs, hepatic AVMs, cerebral AVMs, GI telangiectasias): The inferior mesenteric venous varix is NOT a typical HHT vascular malformation 1, 2
  • First-degree relative with HHT: Not mentioned 1

Diagnostic classification: 1

  • Definite HHT = 3 of 4 criteria
  • Possible/suspected HHT = 2 of 4 criteria
  • Unlikely HHT = fewer than 2 criteria

This patient has at most 1 criterion (and even that is questionable), making HHT unlikely by clinical standards. 1

The ENG Variant of Uncertain Significance

A VUS in ENG is insufficient to diagnose HHT—clinical criteria must be met. 1 The presence of an ENG variant alone does not establish disease, as: 1, 3

  • VUS designation means the variant's pathogenicity is unproven 3
  • Even pathogenic ENG mutations require clinical manifestations for HHT diagnosis 1
  • Approximately 97% of patients with definite clinical HHT (meeting 3+ Curaçao criteria) have identifiable mutations, but the reverse is not true—genetic findings must correlate with phenotype 1, 2

The Venous Varix Is Not an HHT Lesion

The inferior mesenteric venous varix does not represent a typical HHT vascular malformation. 2, 4 HHT is characterized by: 2, 4

  • Arteriovenous malformations (AVMs): Direct artery-to-vein connections bypassing the capillary bed 2, 4
  • Telangiectasias: Enlarged vessels with thin walls prone to rupture 5

A venous varix is a dilated vein without arterial involvement and does not fit the pathophysiology of HHT vascular lesions. 2 This finding should not count toward the visceral lesion criterion. 1

Age and Phenotype Considerations

At age 54, this patient is well beyond the typical age of HHT manifestation: 6

  • Epistaxis typically begins at mean age 11 years in HHT1 (ENG mutations) 6
  • Symptoms generally appear by age 30 6
  • Over 90% of adults with HHT have epistaxis 1

The complete absence of epistaxis at age 54 makes HHT highly unlikely, even with an ENG variant. 1, 6

Recommended Next Steps

Despite the low likelihood of HHT, the presence of an ENG VUS warrants: 1, 6

  1. Detailed family history assessment for HHT features in first-degree relatives (epistaxis, telangiectasias, known HHT diagnosis) 1

  2. Physical examination specifically looking for mucocutaneous telangiectasias on lips, tongue, oral mucosa, fingertips, and nail beds 1, 5

  3. Genetic counseling to evaluate the ENG variant through: 3

    • Review of variant databases for prior HHT associations
    • Functional prediction analysis
    • Segregation analysis if family members are available for testing
  4. If family history or examination reveals additional Curaçao criteria, then proceed with organ-specific screening: 1, 6

    • Contrast echocardiography or chest CT for pulmonary AVMs (more frequent in HHT1/ENG mutations) 6
    • Brain MRI for cerebral AVMs (more common in HHT1) 6
    • Upper endoscopy if unexplained anemia develops 1
  5. Do NOT perform liver biopsy under any circumstances if HHT becomes suspected, due to catastrophic hemorrhage risk 1, 6

Critical Pitfall to Avoid

Never diagnose HHT based on genetic testing alone—the clinical phenotype drives diagnosis. 1 A VUS has uncertain pathogenicity by definition, and even confirmed pathogenic mutations require clinical correlation. 3 This patient's lack of epistaxis, telangiectasias, and true AVMs makes HHT diagnosis untenable regardless of genetic findings. 1, 2

References

Guideline

Diagnostic Approach for Hereditary Hemorrhagic Telangiectasia

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Research

Hereditary hemorrhagic telangiectasia: an overview of diagnosis, management, and pathogenesis.

Genetics in medicine : official journal of the American College of Medical Genetics, 2011

Research

Hereditary hemorrhagic telangiectasia: from molecular biology to patient care.

Journal of thrombosis and haemostasis : JTH, 2010

Guideline

HHT Type 1 Clinical Characteristics and Management

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

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Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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