The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients

Adriana Ceci, Paola Baiardi, Mariagrazia Felisi, Maria Domenica Cappellini, Vittorio Carnelli, Vincenzo De Sanctis, Renzo Galanello, Aurelio Maggio, Giuseppe Masera, Antonio Piga, Francesco Schettini, Ippazio Stefàno, Fernando Tricta

Research output: Contribution to journalArticlepeer-review


In 1997, the Italian Ministry of Health created a special programme for the controlled distribution of deferiprone to collect data and to evaluate its safety and effectiveness in long-term use. Five hundred and thirty-two thalassaemia patients from 86 treatment centres were enrolled in this programme. One hundred and eighty-seven patients (32%) experienced a total of 269 events that led to a temporary interruption or, in some cases, to a discontinuation of treatment. The incidence of agranulocytosis and milder neutropenias were 0.4/100 and 2.1/100 patient-years respectively. Neutropenia occurred predominantly in younger and non-splenectomized patients. Transient alanine transaminase increase, gastrointestinal discomfort and arthralgia were the other most commonly reported events. Ferritin levels showed a significant decrease in time after 3 years of therapy. This is the largest number of deferiprone-treated patients to have been reported to date. These data show that the drug was effective in reducing serum ferritin levels and the incidence of adverse events was not greater than the frequency reported in clinical trials.

Original languageEnglish
Pages (from-to)330-336
Number of pages7
JournalBritish Journal of Haematology
Issue number1
Publication statusPublished - 2002


  • Chelation therapy
  • Deferiprone
  • Effectiveness
  • Safety
  • Thalassaemia major

ASJC Scopus subject areas

  • Hematology


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