{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T13:43:00Z","timestamp":1791294180975,"version":"4.1.0"},"reference-count":21,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2007,10,26]],"date-time":"2007-10-26T00:00:00Z","timestamp":1193356800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2008,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p><jats:bold>Objectives\/methods:\u2002<\/jats:bold> This 1\u2010yr prospective phase II trial evaluated the efficacy of deferasirox in regularly transfused patients aged 3\u201381\u2003yrs with myelodysplastic syndromes (MDS; <jats:italic>n<\/jats:italic>\u2003=\u200347), Diamond\u2013Blackfan anaemia (DBA; <jats:italic>n<\/jats:italic>\u2003=\u200330), other rare anaemias (<jats:italic>n<\/jats:italic>\u2003=\u200322) or \u03b2\u2010thalassaemia (<jats:italic>n<\/jats:italic>\u2003=\u200385). Dosage was determined by baseline liver iron concentration (LIC).<\/jats:p><jats:p><jats:bold>Results:\u2002<\/jats:bold> In patients with baseline LIC\u2003\u22657\u2003mg Fe\/g dry weight, deferasirox initiated at 20 or 30\u2003mg\/kg\/d produced statistically significant decreases in LIC (<jats:italic>P\u2003<\/jats:italic>&lt;\u20030.001); these decreases were greatest in MDS and least in DBA. As chelation efficiency and iron excretion did not differ significantly between disease groups, the differences in LIC changes are consistent with mean transfusional iron intake (least in MDS: 0.28\u2003\u00b1\u20030.14\u2003mg\/kg\/d; greatest in DBA: 0.4\u2003\u00b1\u20030.11\u2003mg\/kg\/d). Overall, LIC changes were dependent on dose (<jats:italic>P\u2003<\/jats:italic>&lt;\u20030.001) and transfusional iron intake (<jats:italic>P\u2003<\/jats:italic>&lt;\u20030.01), but not statistically different between disease groups. Changes in serum ferritin and LIC were correlated irrespective of disease group (<jats:italic>r<\/jats:italic>\u2003=\u20030.59), supporting the potential use of serum ferritin for monitoring deferasirox therapy. Deferasirox had a safety profile compatible with long\u2010term use. There were no disease\u2010specific safety\/tolerability effects: the most common adverse events were gastrointestinal disturbances, skin rash and non\u2010progressive serum creatinine increases.<\/jats:p><jats:p><jats:bold>Conclusions:\u2002<\/jats:bold> Deferasirox is effective for reducing iron burden with a defined, clinically manageable safety profile in patients with various transfusion\u2010dependent anaemias. There were no disease\u2010specific adverse events. Once differences in transfusional iron intake are accounted for, dose\u2010dependent changes in LIC or serum ferritin are similar in MDS and other disease groups.<\/jats:p>","DOI":"10.1111\/j.1600-0609.2007.00985.x","type":"journal-article","created":{"date-parts":[[2007,11,20]],"date-time":"2007-11-20T03:02:08Z","timestamp":1195527728000},"page":"168-176","source":"Crossref","is-referenced-by-count":192,"title":["Relative response of patients with myelodysplastic syndromes and other transfusion\u2010dependent anaemias to deferasirox (ICL670): a 1\u2010yr prospective 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