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This review aims to explore the pathophysiological basis of hereditary hemochromatosis and assess new therapeutic options.

April 3, 2026Future PharmacologyOpen Access

Hereditary Hemochromatosis: Pathophysiological Basis and Emerging Therapeutic Approaches—A Systematic Review of Clinical Evidence

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Systematic review examines emerging therapies for hereditary hemochromatosis, suggesting need for further trials.

This review aims to explore the pathophysiological basis of hereditary hemochromatosis and assess new therapeutic options.
Conducted a systematic review of interventional clinical trials registered on ClinicalTrials.gov.
Included studies with available results regardless of clinical phase or recruitment status.
Extracted and analyzed data on therapeutic interventions and iron metabolism.
Three studies evaluated therapies: hepcidin mimetic Rusfertide, synthetic hepcidin LJPC-401, and iron chelator Deferasirox.
Emerging therapies showed biological activity in modulating iron metabolism parameters but lacked superior efficacy over phlebotomy.
Therapeutic phlebotomy remains the first-line treatment for hereditary hemochromatosis.

Silva et al. (2026) studied this question.

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