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Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non‐transfusion‐dependent thalassaemia |
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| Authors: | Ali T. Taher John B. Porter Vip Viprakasit Antonis Kattamis Suporn Chuncharunee Pranee Sutcharitchan Noppadol Siritanaratkul Raffaella Origa Zeynep Karakas Dany Habr Zewen Zhu Maria Domenica Cappellini |
| Affiliation: | 1. American University of Beirut, Beirut, Lebanon;2. University College London, London, UK;3. Department of Paediatrics and Thalassemia Centre, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand;4. First Department of Paediatrics, University of Athens, Athens, Greece;5. Ramathibodi Hospital, Mahidol University, Bangkok, Thailand;6. Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand;7. Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand;8. Ospedale Regional Microcitemie, Cagliari, Italy;9. Istanbul Medical Faculty, Istanbul University, Istanbul, Turkey;10. Novartis Pharmaceuticals, East Hanover, NJ, USA;11. Università di Milano, Ca Granda Foundation IRCCS, Milan, Italy |
| Abstract: | Liver iron concentration (LIC) assessment by magnetic resonance imaging (MRI) remains the gold standard to diagnose iron overload and guide iron chelation therapy in patients with non‐transfusion‐dependent thalassaemia (NTDT). However, limited access to MRI technology and expertise worldwide makes it practical to also use serum ferritin assessments. The THALASSA (assessment of Exjade® in non‐transfusion‐dependent THALASSemiA patients) study assessed the efficacy and safety of deferasirox in iron‐overloaded NTDT patients and provided a large data set to allow exploration of the relationship between LIC and serum ferritin. Using data from screened patients and those treated with deferasirox for up to 2 years, we identified clinically relevant serum ferritin thresholds (for when MRI is unavailable) for the initiation of chelation therapy (>800 μg/l), as well as thresholds to guide chelator dose interruption (<300 μg/l) and dose escalation (>2000 μg/l). (clinicaltrials.gov identifier: NCT00873041). |
| Keywords: | iron overload chelation therapy non‐transfusion‐dependent thalassaemia liver iron concentration serum ferritin |