The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients.
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.
Ceci
Adriana
A
Dipartimento di Farmacologia, Università di Bari, Fondazione Salvatore Maugeri, Research Institute, Pavia, Italy. aceci@fsm.it
Baiardi
Paola
P
Felisi
Mariagrazia
M
Cappellini
Maria Domenica
MD
Carnelli
Vittorio
V
De Sanctis
Vincenzo
V
Galanello
Renzo
R
Maggio
Aurelio
A
Masera
Giuseppe
G
Piga
Antonio
A
Schettini
Francesco
F
Stefàno
Ippazio
I
Tricta
Fernando
F
D000293:Adolescent; D000328:Adult; D000380:Agranulocytosis / Q000209:etiology; D000410:Alanine Transaminase / Q000097:blood; D001803:Blood Transfusion; D002648:Child; D015331:Cohort Studies; D000077543:Deferiprone; D016903:Drug Monitoring; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007558:Italy; D008297:Male; D008875:Middle Aged; D009503:Neutropenia / Q000209:etiology; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D013156:Splenectomy; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000601:surgery / Q000628:therapy
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D000410:Alanine Transaminase