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The safety and effectiveness of deferiprone in a large‐scale, 3‐year study in Italian patients
Author(s) -
Ceci Adriana,
Baiardi Paola,
Felisi Mariagrazia,
Cappellini Maria Domenica,
Carnelli Vittorio,
De Sanctis Vincenzo,
Galanello Renzo,
Maggio Aurelio,
Masera Giuseppe,
Piga Antonio,
Schettini Francesco,
Stefàno Ippazio,
Tricta Fernando
Publication year - 2002
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.2002.03554.x
Subject(s) - deferiprone , medicine , discontinuation , neutropenia , incidence (geometry) , adverse effect , christian ministry , pediatrics , surgery , thalassemia , chemotherapy , philosophy , physics , theology , optics
Summary. 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.