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Prepublished online as a Blood First Edition Paper on May 22, 2003; DOI 10.1182/blood-2002-10-3280.
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Blood, 1 September 2003, Vol. 102, No. 5, pp. 1583-1587
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC
TRIALS
Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone
Alan R. Cohen,
Renzo Galanello,
Antonio Piga,
Vincenzo De Sanctis, and
Fernando Tricta
From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale
Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie,
Università di Cagliari, Cagliari, Italy; Dipartimento Scienze
Pediatriche e dell'Adolescenza, Università di Torino, Torino, Italy;
Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara,
Italy; and Apotex Research, Toronto, ON, Canada.
The identification of a safe, orally active iron chelator is critically
important for the prevention of morbidity and early death in patients
receiving regular red cell transfusions. Based on our findings in a 1-year
multicenter, prospective study of the safety and efficacy of deferiprone in
patients with thalassemia major, we have extended the treatment period to 4
years. The mean dose of the chelator was 73 mg/kg per day during 531
patient-years. The rates of agranulocytosis (absolute neutrophil count [ANC]
< 500 x 109/L) and milder forms of neutropenia (ANC,
500-1500 x 109/L) were 0.2 and 2.8 per 100 patient-years,
respectively. Neutropenia occurred significantly more commonly in patients
with intact spleens. Gastrointestinal and joint symptoms decreased
significantly after the first year of therapy, and led to discontinuation of
deferiprone in only one patient in years 2 to 4. The mean alanine
aminotransferase (ALT) value of 71 U/L after 4 years of therapy was
significantly higher than the baseline value of 61 U/L. Trend analysis showed
no increase in the ALT levels or the percentage of patients with ALT levels
greater than twice the upper limit of the reference range. Ferritin levels did
not change significantly from the values at the time of change from
deferoxamine to deferiprone in either the intention-to-treat analysis or in
the 84 patients who completed 4 years of therapy. Because of concerns
regarding the effectiveness of the studied dose of deferiprone, 47 patients
discontinued therapy, whereas 15 patients interrupted therapy because of
concerns regarding low iron levels. The results of this study help to define
the safety and effectiveness of long-term therapy with deferiprone.
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