Gastrointestinal · PANCE / PANRE

Hereditary Hemochromatosis

Autosomal recessive HFE-related iron overload → cirrhosis, diabetes, cardiomyopathy, arthropathy, hypogonadism, hyperpigmentation.

Also known as: hereditary hemochromatosis, HH, HFE hemochromatosis, iron overload

Overview

Most commonly an autosomal recessive disorder of iron metabolism due to mutations in HFE (chromosome 6, especially C282Y homozygosity or C282Y/H63D compound heterozygosity), producing inappropriately low hepcidin and consequent excessive intestinal iron absorption. Non-HFE forms (juvenile hemochromatosis, TfR2, ferroportin) are rare.

Epidemiology

Most common genetic disorder in people of Northern European descent — C282Y homozygosity in ~1 in 200-300 individuals; clinical penetrance is much lower (~10-30% in men, lower in women due to menstruation/pregnancy). Symptoms typically emerge after age 40 in men and after menopause in women.

🔒 Free preview limit reached

Keep reading — start your free trial

You've read your 2 free diagnosis previews. Create your free account to unlock the full Hereditary Hemochromatosis outline — plus all 514 diagnoses, 3,500+ board-style questions, flashcards, and an AI tutor. Your 7-day free trial includes everything, and there's no credit card required.

Free to start · No credit card · Cancel anytime

Risk factors

  • C282Y/C282Y homozygosity (~80-90% of clinical cases)
  • C282Y/H63D compound heterozygosity (lower penetrance)
  • Male sex (no menstrual or pregnancy iron losses)
  • Increased dietary iron, iron supplementation, vitamin C supplementation
  • Alcohol use (accelerates hepatic injury)
  • Concomitant hepatitis C, NAFLD, or porphyria cutanea tarda

Pathophysiology

HFE protein normally signals hepcidin synthesis in proportion to iron stores. HFE mutations blunt hepcidin response, allowing unrestrained intestinal absorption of dietary iron. Excess iron deposits as hemosiderin in hepatocytes, pancreatic islets, cardiac myocytes, anterior pituitary, joint synovium, and skin melanocytes, producing oxidative injury and fibrosis in affected organs.

Clinical presentation

Symptoms

  • Fatigue, weakness, malaise (early, nonspecific)
  • Arthralgia, especially 2nd and 3rd MCP joints ('iron fist'); chondrocalcinosis
  • Diabetes ('bronze diabetes' from pancreatic deposition)
  • Hepatic: hepatomegaly, abdominal pain, evolving to cirrhosis with stigmata of chronic liver disease
  • Cardiac: cardiomyopathy (restrictive or dilated), arrhythmias, congestive heart failure
  • Endocrine: hypogonadism (loss of libido, amenorrhea, infertility, erectile dysfunction), hypothyroidism, adrenal insufficiency (rare)
  • Skin: slate-gray or bronze hyperpigmentation (sun-exposed areas)

Signs / physical exam

  • Hepatomegaly with or without splenomegaly
  • Hyperpigmentation
  • Loss of body hair, testicular atrophy
  • Findings of advanced liver disease in late stages
  • MCP joint tenderness, swelling

Classic findings

Classic 'bronze diabetes' tetrad: cirrhosis, diabetes, hyperpigmentation, and hypogonadism in a middle-aged man of Northern European descent.

Differential diagnosis

  • Secondary iron overload — Transfusion-dependent anemias (thalassemia, sickle cell, MDS), repeated transfusions; ferritin high but mutation analysis negative
  • Nonalcoholic fatty liver disease — Metabolic syndrome, steatosis on imaging; iron studies may show modest elevation but not severe overload
  • Alcoholic liver disease — Heavy alcohol use, AST:ALT >2:1, GGT elevation; can coexist and accelerate iron-related injury
  • Wilson disease — Copper, not iron; younger patients, neuropsychiatric features, Kayser-Fleischer rings
  • Porphyria cutanea tarda — Photosensitive vesicles, urine fluoresces under Wood lamp; often coexists with HH
  • Inflammation-related ferritin elevation — Ferritin is an acute-phase reactant; transferrin saturation distinguishes overload from inflammation

Diagnostic workup

Diagnostic criteria

Elevated Tsat and ferritin with C282Y homozygosity (or C282Y/H63D with iron overload) establishes the diagnosis. Liver biopsy if biochemical evidence of overload without typical genotype, or to stage fibrosis.

Labs

  • Fasting transferrin saturation (Tsat) — first-line screening; elevated >45% (women) or >50% (men) suggests iron overload
  • Ferritin — elevated >200 ng/mL (premenopausal women) or >300 ng/mL (men and postmenopausal women); higher levels (>1000) correlate with risk of cirrhosis
  • If both abnormal → HFE genetic testing (C282Y, H63D)
  • LFTs, glucose/A1c, lipid panel
  • TSH, testosterone, FSH/LH if hypogonadism suspected
  • ECG, echocardiogram if cardiac involvement suspected

Imaging

  • MRI with T2* or R2 quantification of liver iron — noninvasive iron quantification, increasingly used over biopsy
  • Liver biopsy with hepatic iron index — historically gold standard; reserved for evaluating fibrosis when ferritin >1000, elevated transaminases, or hepatomegaly

Diagnostic algorithm

flowchart TD
  A[Fatigue + arthralgia + LFT elevation<br/>± diabetes / cardiomyopathy / bronze skin] --> B[Fasting transferrin saturation<br/>+ ferritin]
  B --> C{Tsat >45/50%<br/>and ferritin elevated?}
  C -->|No| D[Consider other diagnoses]
  C -->|Yes| E[HFE genotype<br/>C282Y, H63D]
  E --> F{C282Y/C282Y or<br/>C282Y/H63D with overload?}
  F -->|Yes| G[Hereditary hemochromatosis]
  F -->|No| H[Consider non-HFE / secondary iron overload<br/>± liver biopsy]
  G --> I[Therapeutic phlebotomy<br/>weekly until ferritin 50-100]
  I --> J[Maintenance phlebotomy<br/>+ screen for cirrhosis/HCC<br/>+ family screening]
Diagnostic and treatment algorithm for hereditary hemochromatosis.

Treatment

First-line

  • Therapeutic phlebotomy — mainstay; remove 500 mL whole blood (~250 mg iron) weekly to biweekly until ferritin 50-100 ng/mL and Tsat <50%
  • Maintenance phlebotomy every 2-4 months thereafter to keep ferritin ~50-100
  • Avoid iron and vitamin C supplements; moderate dietary iron (limit red meat, organ meats); avoid raw shellfish (Vibrio vulnificus risk)
  • Limit or avoid alcohol
  • Screen and treat associated conditions: diabetes, hypogonadism (testosterone replacement after iron normalization), cardiomyopathy (standard HF therapy), arthropathy (NSAIDs, joint replacement if severe)

Second-line / adjunct

  • Iron chelation (deferoxamine, deferasirox, deferiprone) — for patients who cannot tolerate phlebotomy (anemia, poor venous access, cardiac decompensation)
  • Erythrocytapheresis — alternative for patients with cardiac compromise
  • Cirrhosis screening: HCC surveillance with ultrasound (± AFP) every 6 months in cirrhotic patients
  • Liver transplantation for decompensated cirrhosis or HCC; iron overload generally improves post-transplant but does not always normalize hepcidin

Complications

  • Cirrhosis (up to 25% of homozygous men eventually)
  • Hepatocellular carcinoma (up to 100-fold relative risk; develops almost exclusively in cirrhotics)
  • Diabetes mellitus
  • Cardiomyopathy and arrhythmias
  • Hypogonadism and infertility
  • Arthropathy and chondrocalcinosis
  • Increased risk of Listeria, Yersinia, Vibrio vulnificus infections (iron-loving organisms)

PANCE pearls

  • Order Tsat AND ferritin together — Tsat alone is more specific; ferritin alone is an acute-phase reactant and easily misleading.
  • Phlebotomy aggressively until ferritin 50-100, then maintenance — do not 'normalize' to mid-range (risk of re-accumulation).
  • Counsel against raw oysters and shellfish — Vibrio vulnificus is highly lethal in iron-overloaded patients.
  • Hepatocellular carcinoma surveillance only if cirrhotic; non-cirrhotic HH does not require routine HCC surveillance.
  • C282Y heterozygotes (single copy) are not at risk for iron overload from HH alone, though minor lab abnormalities can occur in the setting of another liver insult.

References

  • AASLD 2019 — AASLD Practice Guidance on Hemochromatosis (Bacon et al., Hepatology 2011; AASLD 2019 update)
  • EASL 2022 — EASL Clinical Practice Guidelines on haemochromatosis (J Hepatol 2022)
  • ACG 2019 — ACG Clinical Guideline: Hereditary Hemochromatosis (Kowdley et al., Am J Gastroenterol 2019)

Practice Gastrointestinal questions on FirstPassPA

Turn this outline into retention. 3,500+ board-style questions with an AI tutor that explains every answer — free to start, no card required.

Start studying free → Browse all 514 diagnoses

Educational use only. This outline is a study aid for PA students and is not medical advice or a substitute for clinical judgment. FirstPassPA is an independent study tool and is not affiliated with, endorsed by, or sponsored by NCCPA. PANCE® and PANRE® are registered trademarks of the National Commission on Certification of Physician Assistants.