Disease Directory Hereditary Hemochromatosis
Metabolic

Hereditary Hemochromatosis

Also known as: HFE-related hemochromatosis, HFE hereditary hemochromatosis, iron overload disease

Prevalence

1-5 per 1,000 (Orphanet)

Onset

Adult

Type

Autosomal recessive genetic

Gene

HFE

About Hereditary Hemochromatosis

Hereditary hemochromatosis is the most common genetic disorder of iron metabolism in populations of Northern European descent, most often caused by homozygous C282Y mutations in the HFE gene. Unregulated intestinal iron absorption leads to progressive iron deposition in the liver, heart, pancreas, joints, and endocrine glands. Clinical manifestations include liver cirrhosis, cardiomyopathy, diabetes mellitus, arthropathy, and hypogonadism. Phlebotomy (blood removal) remains the cornerstone of treatment.

Common Clinical Features

Liver cirrhosis Fatigue Arthropathy Diabetes mellitus Cardiomyopathy Skin bronzing Hypogonadism

Clinical Trial Eligibility Tips

What to know before applying to Hereditary Hemochromatosis trials.

Serum ferritin and transferrin saturation are the primary eligibility biomarkers — document pre-treatment and current levels

Liver iron concentration by MRI (MRI-LIC) or biopsy is often required for advanced disease trials

Phlebotomy frequency and volume history should be thoroughly documented — some trials require maintenance phase stability

Non-HFE forms (TFR2, HJV, HAMP mutations) are biologically distinct and may have separate or exclusionary eligibility criteria

Patient Resources

Patient Organization

American Hemochromatosis Society

Visit website ↗

Orphanet

European reference resource for rare diseases (ORPHA:93616)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Hereditary Hemochromatosis trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Hereditary Hemochromatosis. Updated daily.

Related Rare Diseases