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Prepublished online as a Blood First Edition Paper on May 22, 2003; DOI 10.1182/blood-2002-10-3280. This Article Full Text Full Text (PDF) All Versions of this Article: 2002-10-3280v1 102/5/1583    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Cohen, A. R. Articles by Tricta, F. Search for Related Content PubMed PubMed Citation Articles by Cohen, A. R. Articles by Tricta, F. Related Collections Red Cells Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  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. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: E. 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