Deferiprone versus Deferoxamine in Patients with Thalassemia Major: A Randomized Clinical Trial

Maggio, Aurelio; D’Amico, Gennaro; Morabito, Alberto; Capra, Marcello; Ciaccio, C; Cianciulli, Paolo; Gregorio, F Di; Garozzo, Giovanni; Malizia, R; Magnano, Carmelo; Mangiagli, A; Quarta, Giovanni; Rizzo, M.; D’Ascola, Domenico Giuseppe; Rizzo, Aroldo; Midiri, Massimo

doi:10.1006/bcmd.2002.0510

Cited by 172 publications

(117 citation statements)

References 31 publications

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“…Despite the high frequency, only a restricted number of patients discontinued therapy because of GI complaints. Similar results have been reported in other studies [24,55,56]. About 5% of patients report increased appetite considered related to DFP, but its significance is unknown.…”

Section: Clinical Safety Profilesupporting

confidence: 90%

“…Results of randomized clinical trials comparing DFO with DFP demonstrated no difference after 12 months of treatment in serum ferritin levels or liver iron concentration [23,24]. Similar results have been obtained in other studies [25,26,27].…”

Section: Dfp Efficacysupporting

confidence: 76%

“…Increase in serum alanine aminotransferase (ALT) activity was reported in about 7% of the patients particularly in the first months of treatment. The transient, asymptomatic, and usually mild changes in ALT levels and the absence of a progressive increase over time have been reported in several studies [24,54,56,58]. However, it is recommend that serum ALT levels be monitored at regular intervals and DFP interruption or dose reduction should be considered if a persistent increase in serum ALT occurs.…”

Section: Clinical Safety Profilementioning

confidence: 97%

“…Several prospective trials have compared the effects of DFP and DFO on myocardial iron. In two studies, DFP at 75 mg/kg/day was as effective as DFO at 50 mg/kg/day for 5–6 days per week at reducing cardiac iron by MR signal intensity ratio [24,32]. Peng et al [27] compared cardiac iron, estimated by MR signal intensity and LVEF over 3 years in 13 patients taking DFO (50 mg/kg/day at least 5 days per week) with 11 patients taking DFP (75 mg/kg/day).…”

Section: Dfp Efficacymentioning

confidence: 99%

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Deferiprone Chelation Therapy for Thalassemia Major

Galanello

Campus²

2009

Acta Haematol

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Iron overload is one of the major causes of morbidity in patients with thalassemia major. Deferiprone (DFP), an orally active iron chelator, emerged from an extensive search for new drugs to treat iron overload. Comparative studies have shown that at comparable doses the efficacy of DFP in removing body iron is similar to that of desferoxamine (DFO). In retrospective and prospective studies, DFP monotherapy was significantly more effective than DFO in the treatment of myocardial siderosis in thalassemia major. DFP can be used in combination with DFO in the management of severe iron overload. This chelation regimen is tolerable and attractive for patients unable to comply with standard DFO infusions or with inadequate response to DFP monotherapy. DFP has a well-known long-term safety profile. Agranulocytosis is the most serious side effect associated with its use, occurring in about 1% of the patients. More common but less serious side effects are gastrointestinal symptoms, arthralgia, zinc deficiency, and fluctuating transaminase levels.

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Section: Clinical Safety Profilesupporting

confidence: 90%

Section: Dfp Efficacysupporting

confidence: 76%

Section: Clinical Safety Profilementioning

confidence: 97%

Section: Dfp Efficacymentioning

confidence: 99%

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Deferiprone Chelation Therapy for Thalassemia Major

Galanello

Campus²

2009

Acta Haematol

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“…Changes in ALT levels were transient and settled without discontinuation of deferiprone therapy. These findings are consistent with the usual transient nature of the changes in ALT levels that has been reported previously [12,14,15,16]. The results of clinical trials with interferon α in hepatitis-C-infected patients with TM suggest that the clinical and histological responses to this agent may be inversely related to body iron burden [17].…”

Section: Discussionsupporting

confidence: 89%

Safety and Effectiveness of 100 mg/kg/day Deferiprone in Patients with Thalassemia Major: A Two-Year Study

et al. 2005

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Deferiprone at a dose of 75 mg/kg/day is not sufficiently effective to maintain iron stores at a level which has been considered safe in all patients with iron overload. Our main aim was to determine the safety of long-term therapy with high-dose (100 mg/kg/day) deferiprone. A secondary aim was to determine the efficacy of this high dose. Twelve thalassemia major patients received deferiprone at a dose of100 mg/kg/day over 2 years. Transient aspartate aminotransferase increase (8 patients), gastrointestinal discomfort (3 patients) and arthralgia (2 patients) were the most commonly reported side effects. None of the patients discontinued therapy. The mean serum ferritin level fell from 3,901 ± 3,618 to 1,790 ± 2,205 µg/l after 2 years (p < 0.05). Five of the 12 patients continued to receive deferiprone for an additional 3 years. No new side effects were encountered. The mean serum ferritin level in this subgroup was initially 2,510 ± 332 µg/l and dropped to 1,511 ± 664 µg/l after 5 years (p < 0.05). Liver iron levels at the end of the 2-year study ranged from 1.0 to 30.9 mg/g dry weight, 3 of the patients having levels above 15 mg/g.

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