Design of an ongoing phase I/II open-label, dose-escalation trial using the oral chelator deferasirox to treat iron overload in HFE-Related hereditary hemochromatosis (HH)
| Autor: | Lisa Rojkjaer, Herbert L. Bonkovsky, Pradyumna D. Phatak, R. Weitzman, S. Bailey, Claus Niederau, Janet Bodner, Antonello Pietrangelo, Pierre Brissot |
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| Předmět: |
medicine.medical_specialty
Transferrin saturation business.industry Immunology Deferasirox Cell Biology Hematology Phlebotomy medicine.disease Biochemistry Gastroenterology Intestinal absorption Surgery Hereditary hemochromatosis Internal medicine medicine Chelation Hemoglobin business Hemochromatosis medicine.drug |
| Zdroj: | Università degli studi di Modena e Reggio Emilia-IRIS ResearcherID |
| Popis: | HH is a genetic disorder commonly associated with homozygosity for the C282Y HFE mutation and characterized by progressive iron overload through increased intestinal absorption. Organ failure due to iron toxicity may develop. Iron removal by phlebotomy is the preferred treatment and has been demonstrated to prevent or reverse some of the complications of iron overload. However, compliance with a weekly phlebotomy schedule is variable, and some patients are ineligible for phlebotomy due to underlying medical disorders. Thus, if an oral iron chelator such as deferasirox proves to be safe and effective, HH patients will have an alternative treatment option. This is an inter-patient dose-escalation study of deferasirox (5, 10, 15, 20 mg/kg) given daily for 24 weeks to C282Y homozygous HH patients with a pre-treatment serum ferritin (SF) value ≥300 μg/L and ≤2000 μg/L, and transferrin saturation ≥45%. Major exclusion criteria are men with hemoglobin Parameter n Mean±SD Median Range Normal range SF, ng/mL 11 633.0±428.9 567.0 350–1880 30–400 (men); 15–150 (women) Transferrin saturation, % 11 75.5±19.6 82.0 39–95 20–55 ALT, U/L 11 43.4±33.4 34.0 8–122 0–45 |
| Databáze: | OpenAIRE |
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