Survival and Causes of Death in Thalassaemia Major

Zurlo, MariaGrazia; Stefano, Piero De; Borgna‐Pignatti, Caterina; Palma, Andrea Di; Melevendi, C; Piga, Antonio; Gregorio, F Di; Burattini, MariaGabriella; Terzoli, S

doi:10.1016/s0140-6736(89)90264-x

Cited by 564 publications

(390 citation statements)

References 18 publications

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“…Cardiac complications are reported to cause 71% of deaths in patients with thalassaemia major [9]. Therefore the heart is the target lethal organ in tha- lassaemia.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of QT dispersion in β thalassaemia major patients

et al. 2006

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Cardiac complications are considered to be the primary cause of death in patients with b thalassaemia major. QT dispersion is a marker variability of ventricular repolarization and is elevated in various high risk groups of patients. This study was carried out in patients with b thalassaemia major to evaluate QT dispersion and to investigate the relationship between QT dispersion and body iron load. Sixty-two b thalassaemia major patients were enrolled into the study. The average serum ferritin levels and liver iron concentration was assessed. For each patient, QTQTc intervals and QT-QTc dispersions were calculated and V1S and V5R were measured. All the subjects underwent two-dimensional M mode echocardiogram and Doppler study. LVMI was found higher in thalassaemia major patients compared to control group. b thalassaemia major patients showed significantly higher mean QT, QTc, QTd, and QTcd values compared to the control group. The mean V5R and V1S amplitudes were also higher in b thalassaemia major patients. There was a positive correlation between LVMI and QTc, QTd and QTcd. However, there was no significant correlation between QT dispersion and serum ferritin and liver iron concentration. Prospective longitudinal studies are needed to assess the prognostic significance of these findings. Am. J. Hematol. 81:901-906, 2006. V V C 2006 Wiley-Liss, Inc.

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“…Cardiac complications are reported to cause 71% of deaths in patients with thalassaemia major [9]. Therefore the heart is the target lethal organ in tha- lassaemia.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of QT dispersion in β thalassaemia major patients

et al. 2006

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“…Splenectomy can be used only in limiting number of patients with hypersplenism and could not ameliorate clinical phenotype in most severe patients. Therefore patients with severe thalassemia syndrome still rely on regular blood transfusion to improve both quality of life [3] and long-term survival [4]. As a direct result, transfusional iron overload is unavoidable and this could cause fatal complications including cardiac siderosis and failure, endocrinopathies, delay puberty, liver fibrosis and failure, and increased susceptibility to infectious diseases [5][6][7].…”

Section: Introductionmentioning

confidence: 99%

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

et al. 2013

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Accessibility to iron chelators including deferoxamine and deferasirox remains obscured in many developing countries. To provide an alternative, the government pharmaceutical organization of Thailand (GPO) manufactured deferiprone which has similar bioequivalent to the standard product. Seventy-three pediatric patients with severe b thalassemias, age range 3.2-19 years, were recruited to a 1-year multicenter prospective, single arm, open label, dose escalating Phase III study of deferiprone to determine its clinical efficacy and safety. Sixty-four patients (87.6%) completed the study with good compliance (>94%). Average deferiprone dose was 79.164.3 mg/kg/day. Overall, mean serum ferritin (SF) levels at 1 year were not significantly changed from baseline. However, 45% of patients (response group) had SF reduced >15% from baseline at 1 year with a median reduction of 1,065 ng ml 21 . Baseline SF was the major factor that predicts clinical efficacy; patients with baseline SF>3,500 ng ml 21 had the most significant fall of SF at 1 year. A subgroup analysis by MRI-T2* confirmed that the response group had higher baseline liver iron and deferiprone could significantly reduce liver iron overload and normalize levels of ALT at 1 year. Although, gastrointestinal irritation (20.5%) was the most common drug-related adverse events (AEs) followed by transaminitis (16.4%) and neutropenia (6.8%), all patients were well tolerated. There was no mortality and agranulocytosis found in this trial. Monotherapy of deferiprone with appropriate dose adjustment and monitoring for adverse events appeared to be an effective chelation therapy in some patients with good compliance and acceptable safety profiles. Am. J. Hematol. 88:251-260,

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“…A major concern with this therapy is the development of transfusional iron overload, which can cause endocrinopathies, liver inflammation and fibrosis, and cardiac toxicity [3][4][5], although some of these complications appear to occur less commonly in SCA compared with thalassemia patients [6,7]. The use of modified transfusion programs that allow higher pretransfusion hemoglobin S levels [8] and automated exchange transfusions may limit iron loading in SCA [9,10].…”

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confidence: 99%

Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention

et al. 2011

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Survival and Causes of Death in Thalassaemia Major

Cited by 564 publications

References 18 publications

Evaluation of QT dispersion in β thalassaemia major patients

Evaluation of QT dispersion in β thalassaemia major patients

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention

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Survival and Causes of Death in Thalassaemia Major

Cited by 564 publications

References 18 publications

Evaluation of QT dispersion in β thalassaemia major patients

Evaluation of QT dispersion in β thalassaemia major patients

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention

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Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand