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 journalArticle

177 Citations (Scopus)

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 languageEnglish
Pages (from-to)330-336
Number of pages7
JournalBritish Journal of Haematology
Volume118
Issue number1
DOIs
Publication statusPublished - 2002

Fingerprint

Safety
Ferritins
Neutropenia
Agranulocytosis
Thalassemia
Incidence
Arthralgia
Alanine Transaminase
Therapeutics
deferiprone
Clinical Trials
Health
Serum
Pharmaceutical Preparations

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 journalArticle

Ceci, A, Baiardi, P, Felisi, M, Cappellini, MD, Carnelli, V, De Sanctis, V, Galanello, R, Maggio, A, Masera, G, Piga, A, Schettini, F, Stefàno, I & Tricta, F 2002, 'The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients', British Journal of Haematology, vol. 118, no. 1, pp. 330-336. https://doi.org/10.1046/j.1365-2141.2002.03554.x
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. / The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. In: British Journal of Haematology. 2002 ; Vol. 118, No. 1. pp. 330-336.
@article{318e20750d80408dbf6ff7c3a1c7b90d,
title = "The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients",
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.",
keywords = "Chelation therapy, Deferiprone, Effectiveness, Safety, Thalassaemia major",
author = "Adriana Ceci and Paola Baiardi and Mariagrazia Felisi and Cappellini, {Maria Domenica} and Vittorio Carnelli and {De Sanctis}, Vincenzo and Renzo Galanello and Aurelio Maggio and Giuseppe Masera and Antonio Piga and Francesco Schettini and Ippazio Stef{\`a}no and Fernando Tricta",
year = "2002",
doi = "10.1046/j.1365-2141.2002.03554.x",
language = "English",
volume = "118",
pages = "330--336",
journal = "British Journal of Haematology",
issn = "0007-1048",
publisher = "John Wiley & Sons, Ltd (10.1111)",
number = "1",

}

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 -