What is the diagnosis and treatment for hereditary hemochromatosis (HH)?

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From the Guidelines

The primary treatment for hemochromatosis is regular phlebotomy, typically starting with weekly sessions removing 500ml of blood until iron levels normalize, then maintenance phlebotomies every 2-4 months, as recommended by the American Association for the Study of Liver Diseases in 2011 1.

Treatment Approach

The treatment approach for hemochromatosis involves:

  • Regular phlebotomy to remove excess iron from the body
  • Checking hematocrit/hemoglobin prior to each phlebotomy to avoid a drop of more than 20% of the prior level
  • Monitoring serum ferritin levels every 10-12 phlebotomies to adjust the treatment plan
  • Stopping frequent phlebotomy when serum ferritin reaches 50-100 μg/L and continuing phlebotomy at intervals to maintain serum ferritin between 50 and 100 μg/L
  • Avoiding vitamin C supplements, as they can enhance iron absorption

Secondary Iron Overload

For patients with secondary iron overload due to dyserythropoiesis, treatment options include:

  • Deferoxamine (Desferal) at a dose of 20-40 mg/kg body weight per day
  • Deferasirox (Exjade) given orally
  • Consideration of follow-up liver biopsy to assess the adequacy of iron removal

Importance of Early Diagnosis and Treatment

Early diagnosis and treatment of hemochromatosis are crucial to prevent complications such as liver cirrhosis, diabetes, heart problems, and arthritis, as emphasized in a study published in 2001 1. This study highlights the importance of instituting phlebotomy therapy before cirrhosis and/or diabetes develop to significantly reduce morbidity and mortality.

Key Considerations

Key considerations in the management of hemochromatosis include:

  • Avoiding iron supplements and vitamin C with meals
  • Limiting alcohol consumption to prevent increased iron absorption and liver damage
  • Considering iron chelation therapy for patients who cannot undergo phlebotomy
  • Monitoring for cardiac dysrhythmias and cardiomyopathy, which are common causes of sudden death in iron overload states.

From the Research

Definition and Diagnosis of Hemochromatosis

  • Hemochromatosis is a genetic disorder that can produce systemic iron deposition leading to end-organ failure and death if untreated 2.
  • The diagnosis of hemochromatosis can be challenging, as elevated serum ferritin may be seen in various conditions, including acute and chronic liver disease, systemic inflammatory states, and primary and secondary iron overload syndromes 2.
  • Laboratory testing for serum ferritin and transferrin saturation can help recognize hemochromatosis, and genetic testing for the HFE mutation can be performed in patients with elevated iron indices and a suspicion for hemochromatosis or liver disease 3.

Treatment and Management of Hemochromatosis

  • Phlebotomy is a common treatment for hemochromatosis, aiming to remove excess iron stores 4, 3.
  • The mean corpuscular volume (MCV) can be used as a guide to the pace of phlebotomy therapy, with changes in MCV and hemoglobin (Hb) used to guide the pace of phlebotomy 4.
  • Deferasirox, an iron chelator, has been shown to be effective in reducing iron burden in patients with hereditary hemochromatosis and may be a safe alternative to phlebotomy in selected patients 5.

Screening and Prevention of Hemochromatosis

  • Screening primary care patients for hereditary hemochromatosis with transferrin saturation and serum ferritin level has been studied, but the available evidence does not demonstrate that benefits outweigh the risks and costs of screening 6.
  • Therapeutic phlebotomy for hereditary hemochromatosis is relatively safe and presumably efficacious when offered before cirrhosis develops, making early diagnosis and treatment crucial 6.

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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