In hereditary hemochromatosis, phlebotomy is the standard first-line intervention to reduce excess iron. When it fails to bring serum ferritin into the target range — or when it cannot be tolerated — a different therapeutic approach is required.
Phlebotomy did not achieve the goal serum ferritin level of 50–100 ng/mL, or the patient was unable to tolerate phlebotomy. This failure to meet the first-line target is the clinical trigger for escalation to the next therapeutic step.
For patients who are intolerant of or refractory to phlebotomy, current evidence supports the use of iron chelation therapy — a class of treatment available in both oral and parenteral forms — to progressively reduce the body's iron burden. The choice of specific agent depends on individual clinical factors and is detailed in the full protocol.
The primary clinical targets are reduction in serum ferritin levels and hepatic iron concentration.
DOI: 10.14309/ajg.0000000000000315
We recommend the use of iron chelation for the treatment of HH for the patient who is intolerant or refractory to phlebotomy or when phlebotomy has the potential for harm, such as in patients with severe anemia or congestive heart failure (strong recommendation, low quality of evidence).
After 12 months of treatment, deferasirox achieved reduction in median ferritin levels and HIC and was well tolerated (148).
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