Abstract
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 language | English |
|---|---|
| Pages (from-to) | 330-336 |
| Number of pages | 7 |
| Journal | British Journal of Haematology |
| Volume | 118 |
| Issue number | 1 |
| DOIs | |
| Publication status | Published - 2002 |
Fingerprint
Keywords
- Chelation therapy
- Deferiprone
- Effectiveness
- Safety
- Thalassaemia major
ASJC Scopus subject areas
- Hematology
Cite this
The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. / 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.
In: British Journal of Haematology, Vol. 118, No. 1, 2002, p. 330-336.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients
AU - Ceci, Adriana
AU - Baiardi, Paola
AU - Felisi, Mariagrazia
AU - Cappellini, Maria Domenica
AU - Carnelli, Vittorio
AU - De Sanctis, Vincenzo
AU - Galanello, Renzo
AU - Maggio, Aurelio
AU - Masera, Giuseppe
AU - Piga, Antonio
AU - Schettini, Francesco
AU - Stefàno, Ippazio
AU - Tricta, Fernando
PY - 2002
Y1 - 2002
N2 - 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.
AB - 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.
KW - Chelation therapy
KW - Deferiprone
KW - Effectiveness
KW - Safety
KW - Thalassaemia major
UR - http://www.scopus.com/inward/record.url?scp=0036068230&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0036068230&partnerID=8YFLogxK
U2 - 10.1046/j.1365-2141.2002.03554.x
DO - 10.1046/j.1365-2141.2002.03554.x
M3 - Article
VL - 118
SP - 330
EP - 336
JO - British Journal of Haematology
JF - British Journal of Haematology
SN - 0007-1048
IS - 1
ER -