LONG-TERM TRIAL OF DEFERIPRONE IN 51 TRANSFUSION-DEPENDENT IRON-OVERLOADED PATIENTS

Fifty-one transfusion-dependent iron-loaded adult patients (38 with th alassemia major) were treated with the orally active iron chelator def eriprone (1,2 dimethyl-3-hydroxypyrid-4-one, L1) at a dose of 75 mg/kg /d (range, 50 to 79). Twenty patients discontinued the drug and five d ied after a mean of 18.7 months (range, 4 to 35). Of the 20, 5 had art hropathy, 5 had gastrointestinal symptoms, 4 had a rising serum ferrit in, 3 had agranulocytosis or neutropenia, 1 had tachycardia, 1 had ren al failure, and 1 went abroad. Twenty-six patients continued deferipro ne for a mean of 39.4 months (range, 12 to 49). Among these patients, there was no overall significant change in serum ferritin !initial mea n, 2,937 mu g/L; range, 980 to 5,970; final mean, 2,323 mu g/L; range, 825 to 5,970) or in urine iran excretion (initial mean, 31.2 mg/24 h; range, 16.3 to 58.2; final mean, 32.1 mg/24 h; range, 9.4 to 75.8), i mplying no overall change in iron stores. When the patients who had re ceived deferiprone for longer than 3 years were considered separately, there was also no significant change in serum ferritin or urinary iro n excretion. The initial serum ferritin levels in the 26 patients who continued deferiprone treatment were significantly lower than in those who discontinued the drug (P <.01). The liver iron content in 17 pati ents who had received deferiprone for 24 to 48 months ranged from 5,9 to 41.2 mg/g dry weight, 50% having levels above 15.0 mg, a level asso ciated with a high risk of cardiac disease due to iron overload. In th is study the drug caused fewer side effects and was more effective at maintaining iron status among patients previously well chelated and wi th lower initial serum ferritin revels. (C) 1998 by The American Socie ty of Hematology.