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Dudley J Pennell - One of the best experts on this subject based on the ideXlab platform.
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international survey of t2 cardiovascular magnetic resonance in β Thalassemia Major
Haematologica, 2013Co-Authors: John Paul Carpenter, Dudley J Pennell, Michael Roughton, Paul Kirk, Lisa J Anderson, John V B Porter, Malcolm J Walker, Renzo Galanello, Fabrice Danjou, Gian Luca ForniAbstract:Accumulation of myocardial iron is the cause of heart failure and early death in most transfused Thalassemia Major patients. T2* cardiovascular magnetic resonance provides calibrated, reproducible measurements of myocardial iron. However, there are few data regarding myocardial iron loading and its relation to outcome across the world. A survey is reported of 3,095 patients in 27 worldwide centers using T2* cardiovascular magnetic resonance. Data on baseline T2* and numbers of patients with symptoms of heart failure at first scan (defined as symptoms and signs of heart failure with objective evidence of left ventricular dysfunction) were requested together with more detailed information about patients who subsequently developed heart failure or died. At first scan, 20.6% had severe myocardial iron (T2*≤ 10 ms), 22.8% had moderate myocardial iron (T2* 10-20 ms) and 56.6% of patients had no iron loading (T2*>20 ms). There was significant geographical variation in myocardial iron loading (24.8-52.6%; P<0.001). At first scan, 85 (2.9%) of 2,915 patients were reported to have heart failure (81.2% had T2* <10 ms; 98.8% had T2* <20 ms). During follow up, 108 (3.8%) of 2,830 patients developed new heart failure. Of these, T2* at first scan had been less than 10 ms in 96.3% and less than 20 ms in 100%. There were 35 (1.1%) cardiac deaths. Of these patients, myocardial T2* at first scan had been less than 10 ms in 85.7% and less than 20 ms in 97.1%. Therefore, in this worldwide cohort of Thalassemia Major patients, over 43% had moderate/severe myocardial iron loading with significant geographical differences, and myocardial T2* values less than 10 ms were strongly associated with heart failure and death.
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cardiovascular function and treatment in β Thalassemia Major a consensus statement from the american heart association
Circulation, 2013Co-Authors: Dudley J Pennell, Renzo Galanello, James E Udelson, Andrew E Arai, Biykem Bozkurt, Alan R Cohen, Timothy M Hoffman, Michael S Kiernan, Stamatios Lerakis, Antonio PigaAbstract:This aim of this statement is to report an expert consensus on the diagnosis and treatment of cardiac dysfunction in β-Thalassemia Major (TM). This consensus statement does not cover other hemoglob...
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cardiac t2 magnetic resonance for prediction of cardiac complications in Thalassemia Major
Circulation, 2009Co-Authors: Paul Kirk, Mark A Tanner, Mark Westwood, Michael Roughton, Lisa J Anderson, John B Porter, J M Walker, Junaid Patel, J Taylor, Dudley J PennellAbstract:Background— The goal of this study was to determine the predictive value of cardiac T2* magnetic resonance for heart failure and arrhythmia in Thalassemia Major. Methods and Results— We analyzed cardiac and liver T2* magnetic resonance and serum ferritin in 652 Thalassemia Major patients from 21 UK centers with 1442 magnetic resonance scans. The relative risk for heart failure with cardiac T2* values 10 ms) was 160 (95% confidence interval, 39 to 653). Heart failure occurred in 47% of patients within 1 year of a cardiac T2* 20 ms) was 4.6 (95% conf...
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combined chelation therapy in Thalassemia Major for the treatment of severe myocardial siderosis with left ventricular dysfunction
Journal of Cardiovascular Magnetic Resonance, 2008Co-Authors: Mark A Tanner, Annalisa Agus, Mark Westwood, Carlo Dessi, Gillian C Smith, Malcolm J Walker, Sunil V Nair, Martina Pibiri, Dudley J PennellAbstract:Background In Thalassemia Major (TM), severe cardiac siderosis can be treated by continuous parenteral deferoxamine, but poor compliance, complications and deaths occur. Combined chelation therapy with deferiprone and deferoxamine is effective for moderate myocardial siderosis, but has not been prospectively examined in severe myocardial siderosis.
Gian Luca Forni - One of the best experts on this subject based on the ideXlab platform.
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international survey of t2 cardiovascular magnetic resonance in β Thalassemia Major
Haematologica, 2013Co-Authors: John Paul Carpenter, Dudley J Pennell, Michael Roughton, Paul Kirk, Lisa J Anderson, John V B Porter, Malcolm J Walker, Renzo Galanello, Fabrice Danjou, Gian Luca ForniAbstract:Accumulation of myocardial iron is the cause of heart failure and early death in most transfused Thalassemia Major patients. T2* cardiovascular magnetic resonance provides calibrated, reproducible measurements of myocardial iron. However, there are few data regarding myocardial iron loading and its relation to outcome across the world. A survey is reported of 3,095 patients in 27 worldwide centers using T2* cardiovascular magnetic resonance. Data on baseline T2* and numbers of patients with symptoms of heart failure at first scan (defined as symptoms and signs of heart failure with objective evidence of left ventricular dysfunction) were requested together with more detailed information about patients who subsequently developed heart failure or died. At first scan, 20.6% had severe myocardial iron (T2*≤ 10 ms), 22.8% had moderate myocardial iron (T2* 10-20 ms) and 56.6% of patients had no iron loading (T2*>20 ms). There was significant geographical variation in myocardial iron loading (24.8-52.6%; P<0.001). At first scan, 85 (2.9%) of 2,915 patients were reported to have heart failure (81.2% had T2* <10 ms; 98.8% had T2* <20 ms). During follow up, 108 (3.8%) of 2,830 patients developed new heart failure. Of these, T2* at first scan had been less than 10 ms in 96.3% and less than 20 ms in 100%. There were 35 (1.1%) cardiac deaths. Of these patients, myocardial T2* at first scan had been less than 10 ms in 85.7% and less than 20 ms in 97.1%. Therefore, in this worldwide cohort of Thalassemia Major patients, over 43% had moderate/severe myocardial iron loading with significant geographical differences, and myocardial T2* values less than 10 ms were strongly associated with heart failure and death.
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cardiac morbidity and mortality in deferoxamine or deferiprone treated patients with Thalassemia Major
Blood, 2006Co-Authors: Caterina Borgnapignatti, Piero De Stefano, Giovanni Carlo Del Vecchio, Roberta Ghilardi, M A Romeo, Maria Rita Gamberini, Gian Luca Forni, Maria Domenica Cappellini, Antonio Piga, Huaqing ZhaoAbstract:Deferoxamine (DFO) therapy has been associated with improved survival of Thalassemia patients. However, cardiac disease remains the main cause of death in those patients. In 1995, the oral chelator deferiprone became available for clinical use. We compared the occurrence of cardiac disease in patients treated only with DFO and in those whose therapy was switched to deferiprone during the period of observation, from January 31, 1995, to December 31, 2003. All patients with Thalassemia Major treated in 7 Italian centers who were born between 1970 and 1993 and who had not experienced a cardiac event prior to January 1995 were included. DFO only was given to 359 patients, and 157 patients received deferiprone for part of the time. A total of 3610 patient-years were observed on DFO and 750 on deferiprone. At baseline, the 2 groups were comparable for age and sex, while ferritin levels were significantly higher in patients switched to deferiprone. Fifty-two cardiac events, including 10 cardiac deaths, occurred during therapy with DFO. No cardiac events occurred during deferiprone therapy or within at least 18 months after the end of it. In the setting of a natural history study, deferiprone therapy was associated with significantly greater cardiac protection than deferoxamine in patients with Thalassemia Major.
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survival and complications in patients with Thalassemia Major treated with transfusion and deferoxamine
Haematologica, 2004Co-Authors: Caterina Borgnapignatti, Piero De Stefano, M A Romeo, Maria Rita Gamberini, Gian Luca Forni, Huaqing Zhao, Maria Domenica Cappellini, Simone Rugolotto, Giovanni Carlo Del Vecchio, Roberta GhilardiAbstract:BACKGROUND AND OBJECTIVES: Seven Italian centers reported data on survival, causes of death and appearance of complications in patients with Thalassemia Major. The interactions between gender, birth cohort, complications, and ferritin on survival and complications were analyzed. DESIGN AND METHODS: Survival after the first decade was studied for 977 patients born since 1960 whereas survival since birth and complication appearance was studied for the 720 patients born after 1970. Better survival was demonstrated for patients born in more recent years (p<0.00005) and for females (p=0.0003); 68% of the patients are alive at the age of 35 years. In the entire population 67% of the deaths were due to heart disease. RESULTS: There was a significant association between birth cohort and complication-free survival (p<0.0005). The prevalence of complications was: heart failure 6.8%, arrhythmia 5.7%, hypogonadism 54.7%, hypothyroidism 10.8%, diabetes 6.4%, HIV infection 1.7%, and thrombosis 1.1%. Lower ferritin levels were associated with a lower probability of heart failure (hazard ratio =3.35, p<0.005) and with prolonged survival (hazard ratio = 2.45, p<0.005), using a cut-off as low as 1,000 ng/mL. INTERPRETATION AND CONCLUSIONS: Survival and complication-free survival of patients with Thalassemia Major continue to improve, especially for female patients born shortly before or after the availability of iron chelation.
Maria Rita Gamberini - One of the best experts on this subject based on the ideXlab platform.
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multiparametric cardiac magnetic resonance survey in children with Thalassemia Major a multicenter study
Circulation-cardiovascular Imaging, 2015Co-Authors: Maddalena Casale, Maria Rita Gamberini, Antonella Meloni, Aldo Filosa, Liana Cuccia, Vincenzo Caruso, Giovanni Palazzi, Lorella Pitrolo, Maria Caterina Putti, Domenico Giuseppe DascolaAbstract:BACKGROUND: Cardiovascular magnetic resonance (CMR) plays a key role in the management of Thalassemia Major patients, but few data are available in pediatric population. This study aims at a retrospective multiparametric CMR assessment of myocardial iron overload, function, and fibrosis in a cohort of pediatric Thalassemia Major patients. METHODS AND RESULTS: We studied 107 pediatric Thalassemia Major patients (61 boys, median age 14.4 years). Myocardial and liver iron overload were measured by T2* multiecho technique. Atrial dimensions and biventricular function were quantified by cine images. Late gadolinium enhancement images were acquired to detect myocardial fibrosis. All scans were performed without sedation. The 21.4% of the patients showed a significant myocardial iron overload correlated with lower compliance to chelation therapy (P<0.013). Serum ferritin ≥2000 ng/mL and liver iron concentration ≥14 mg/g/dw were detected as the best threshold for predicting cardiac iron overload (P=0.001 and P<0.0001, respectively). A homogeneous pattern of myocardial iron overload was associated with a negative cardiac remodeling and significant higher liver iron concentration (P<0.0001). Myocardial fibrosis by late gadolinium enhancement was detected in 15.8% of the patients (youngest children 13 years old). It was correlated with significant lower heart T2* values (P=0.022) and negative cardiac remodeling indexes. A pathological magnetic resonance imaging liver iron concentration was found in the 77.6% of the patients. CONCLUSIONS: Cardiac damage detectable by a multiparametric CMR approach can occur early in Thalassemia Major patients. So, the first T2* CMR assessment should be performed as early as feasible without sedation to tailor the chelation treatment. Conversely, late gadolinium enhancement CMR should be postponed in the teenager age.
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cardiac morbidity and mortality in deferoxamine or deferiprone treated patients with Thalassemia Major
Blood, 2006Co-Authors: Caterina Borgnapignatti, Piero De Stefano, Giovanni Carlo Del Vecchio, Roberta Ghilardi, M A Romeo, Maria Rita Gamberini, Gian Luca Forni, Maria Domenica Cappellini, Antonio Piga, Huaqing ZhaoAbstract:Deferoxamine (DFO) therapy has been associated with improved survival of Thalassemia patients. However, cardiac disease remains the main cause of death in those patients. In 1995, the oral chelator deferiprone became available for clinical use. We compared the occurrence of cardiac disease in patients treated only with DFO and in those whose therapy was switched to deferiprone during the period of observation, from January 31, 1995, to December 31, 2003. All patients with Thalassemia Major treated in 7 Italian centers who were born between 1970 and 1993 and who had not experienced a cardiac event prior to January 1995 were included. DFO only was given to 359 patients, and 157 patients received deferiprone for part of the time. A total of 3610 patient-years were observed on DFO and 750 on deferiprone. At baseline, the 2 groups were comparable for age and sex, while ferritin levels were significantly higher in patients switched to deferiprone. Fifty-two cardiac events, including 10 cardiac deaths, occurred during therapy with DFO. No cardiac events occurred during deferiprone therapy or within at least 18 months after the end of it. In the setting of a natural history study, deferiprone therapy was associated with significantly greater cardiac protection than deferoxamine in patients with Thalassemia Major.
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survival and complications in patients with Thalassemia Major treated with transfusion and deferoxamine
Haematologica, 2004Co-Authors: Caterina Borgnapignatti, Piero De Stefano, M A Romeo, Maria Rita Gamberini, Gian Luca Forni, Huaqing Zhao, Maria Domenica Cappellini, Simone Rugolotto, Giovanni Carlo Del Vecchio, Roberta GhilardiAbstract:BACKGROUND AND OBJECTIVES: Seven Italian centers reported data on survival, causes of death and appearance of complications in patients with Thalassemia Major. The interactions between gender, birth cohort, complications, and ferritin on survival and complications were analyzed. DESIGN AND METHODS: Survival after the first decade was studied for 977 patients born since 1960 whereas survival since birth and complication appearance was studied for the 720 patients born after 1970. Better survival was demonstrated for patients born in more recent years (p<0.00005) and for females (p=0.0003); 68% of the patients are alive at the age of 35 years. In the entire population 67% of the deaths were due to heart disease. RESULTS: There was a significant association between birth cohort and complication-free survival (p<0.0005). The prevalence of complications was: heart failure 6.8%, arrhythmia 5.7%, hypogonadism 54.7%, hypothyroidism 10.8%, diabetes 6.4%, HIV infection 1.7%, and thrombosis 1.1%. Lower ferritin levels were associated with a lower probability of heart failure (hazard ratio =3.35, p<0.005) and with prolonged survival (hazard ratio = 2.45, p<0.005), using a cut-off as low as 1,000 ng/mL. INTERPRETATION AND CONCLUSIONS: Survival and complication-free survival of patients with Thalassemia Major continue to improve, especially for female patients born shortly before or after the availability of iron chelation.
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survival and disease complications in Thalassemia Major
Annals of the New York Academy of Sciences, 1998Co-Authors: Caterina Borgnapignatti, Piero De Stefano, Maria Rita Gamberini, Maria Domenica Cappellini, Antonio Piga, Simone Rugolotto, Felicia Di Gregorio, V Sabato, C Melevendi, Giuseppe VerlatoAbstract:We studied survival and disease complications in 1,146 patients with Thalassemia Major, born from January 1, 1960 to December 31, 1987. At last follow-up, in March 1997, probability of survival to age 20 years was 89% and to age 25 years was 82% for patients born in the years 1970-1974. Patients who died had a serum ferritin level, measured the year before death, significantly higher than those who survived. Diabetes was present in 5.4% of the patients; heart failure in 6.4%; arrhythmias in 5.0%, thrombosis in 1.1%, hypothyroidism in 11.6%, HIV infection in 1.8%. Hypogonadism was diagnosed in 55% of 578 patients who had reached pubertal age: 83.5% of hypogonadic females and 78.6% of males were receiving substitutive hormonal therapy. In conclusion, the survival of patients with Thalassemia Major is good and improving, but the prevalence of severe complications is still high.
Paul Kirk - One of the best experts on this subject based on the ideXlab platform.
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international survey of t2 cardiovascular magnetic resonance in β Thalassemia Major
Haematologica, 2013Co-Authors: John Paul Carpenter, Dudley J Pennell, Michael Roughton, Paul Kirk, Lisa J Anderson, John V B Porter, Malcolm J Walker, Renzo Galanello, Fabrice Danjou, Gian Luca ForniAbstract:Accumulation of myocardial iron is the cause of heart failure and early death in most transfused Thalassemia Major patients. T2* cardiovascular magnetic resonance provides calibrated, reproducible measurements of myocardial iron. However, there are few data regarding myocardial iron loading and its relation to outcome across the world. A survey is reported of 3,095 patients in 27 worldwide centers using T2* cardiovascular magnetic resonance. Data on baseline T2* and numbers of patients with symptoms of heart failure at first scan (defined as symptoms and signs of heart failure with objective evidence of left ventricular dysfunction) were requested together with more detailed information about patients who subsequently developed heart failure or died. At first scan, 20.6% had severe myocardial iron (T2*≤ 10 ms), 22.8% had moderate myocardial iron (T2* 10-20 ms) and 56.6% of patients had no iron loading (T2*>20 ms). There was significant geographical variation in myocardial iron loading (24.8-52.6%; P<0.001). At first scan, 85 (2.9%) of 2,915 patients were reported to have heart failure (81.2% had T2* <10 ms; 98.8% had T2* <20 ms). During follow up, 108 (3.8%) of 2,830 patients developed new heart failure. Of these, T2* at first scan had been less than 10 ms in 96.3% and less than 20 ms in 100%. There were 35 (1.1%) cardiac deaths. Of these patients, myocardial T2* at first scan had been less than 10 ms in 85.7% and less than 20 ms in 97.1%. Therefore, in this worldwide cohort of Thalassemia Major patients, over 43% had moderate/severe myocardial iron loading with significant geographical differences, and myocardial T2* values less than 10 ms were strongly associated with heart failure and death.
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cardiac t2 magnetic resonance for prediction of cardiac complications in Thalassemia Major
Circulation, 2009Co-Authors: Paul Kirk, Mark A Tanner, Mark Westwood, Michael Roughton, Lisa J Anderson, John B Porter, J M Walker, Junaid Patel, J Taylor, Dudley J PennellAbstract:Background— The goal of this study was to determine the predictive value of cardiac T2* magnetic resonance for heart failure and arrhythmia in Thalassemia Major. Methods and Results— We analyzed cardiac and liver T2* magnetic resonance and serum ferritin in 652 Thalassemia Major patients from 21 UK centers with 1442 magnetic resonance scans. The relative risk for heart failure with cardiac T2* values 10 ms) was 160 (95% confidence interval, 39 to 653). Heart failure occurred in 47% of patients within 1 year of a cardiac T2* 20 ms) was 4.6 (95% conf...
Renzo Galanello - One of the best experts on this subject based on the ideXlab platform.
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international survey of t2 cardiovascular magnetic resonance in β Thalassemia Major
Haematologica, 2013Co-Authors: John Paul Carpenter, Dudley J Pennell, Michael Roughton, Paul Kirk, Lisa J Anderson, John V B Porter, Malcolm J Walker, Renzo Galanello, Fabrice Danjou, Gian Luca ForniAbstract:Accumulation of myocardial iron is the cause of heart failure and early death in most transfused Thalassemia Major patients. T2* cardiovascular magnetic resonance provides calibrated, reproducible measurements of myocardial iron. However, there are few data regarding myocardial iron loading and its relation to outcome across the world. A survey is reported of 3,095 patients in 27 worldwide centers using T2* cardiovascular magnetic resonance. Data on baseline T2* and numbers of patients with symptoms of heart failure at first scan (defined as symptoms and signs of heart failure with objective evidence of left ventricular dysfunction) were requested together with more detailed information about patients who subsequently developed heart failure or died. At first scan, 20.6% had severe myocardial iron (T2*≤ 10 ms), 22.8% had moderate myocardial iron (T2* 10-20 ms) and 56.6% of patients had no iron loading (T2*>20 ms). There was significant geographical variation in myocardial iron loading (24.8-52.6%; P<0.001). At first scan, 85 (2.9%) of 2,915 patients were reported to have heart failure (81.2% had T2* <10 ms; 98.8% had T2* <20 ms). During follow up, 108 (3.8%) of 2,830 patients developed new heart failure. Of these, T2* at first scan had been less than 10 ms in 96.3% and less than 20 ms in 100%. There were 35 (1.1%) cardiac deaths. Of these patients, myocardial T2* at first scan had been less than 10 ms in 85.7% and less than 20 ms in 97.1%. Therefore, in this worldwide cohort of Thalassemia Major patients, over 43% had moderate/severe myocardial iron loading with significant geographical differences, and myocardial T2* values less than 10 ms were strongly associated with heart failure and death.
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cardiovascular function and treatment in β Thalassemia Major a consensus statement from the american heart association
Circulation, 2013Co-Authors: Dudley J Pennell, Renzo Galanello, James E Udelson, Andrew E Arai, Biykem Bozkurt, Alan R Cohen, Timothy M Hoffman, Michael S Kiernan, Stamatios Lerakis, Antonio PigaAbstract:This aim of this statement is to report an expert consensus on the diagnosis and treatment of cardiac dysfunction in β-Thalassemia Major (TM). This consensus statement does not cover other hemoglob...
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increased survival and reversion of iron induced cardiac disease in patients with Thalassemia Major receiving intensive combined chelation therapy as compared to desferoxamine alone
Blood Cells Molecules and Diseases, 2010Co-Authors: Maria Eliana Lai, Patrizio Bina, Robert W Grady, Stefania Vacquer, Alessia Pepe, Maria Paola Carta, Franco Sau, Paolo Cianciulli, Aurelio Maggio, Renzo GalanelloAbstract:Abstract Myocardial iron overload is the leading cause of death in patients with β-Thalassemia Major. An intensification monotherapy with deferoxamine (DFO) as well as a combination therapy with DFO and deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for Thalassemia Major patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight Thalassemia Major patients with cardiac disease were enrolled in a prospective study lasting 42 ± 6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40–50 mg/kg over 8–12 h at night 5–7 days/week), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events ( p = 0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in Thalassemia Major patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.
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deferiprone chelation therapy for Thalassemia Major
Acta Haematologica, 2009Co-Authors: Renzo Galanello, S CampusAbstract:Iron overload is one of the Major causes of morbidity in patients with Thalassemia Major. Deferiprone (DFP), an orally active iron chelator, emerged from an extensive search for new drugs to treat iron overload. Comparative studies have shown that at comparable doses the efficacy of DFP in removing body iron is similar to that of desferoxamine (DFO). In retrospective and prospective studies, DFP monotherapy was significantly more effective than DFO in the treatment of myocardial siderosis in Thalassemia Major. DFP can be used in combination with DFO in the management of severe iron overload. This chelation regimen is tolerable and attractive for patients unable to comply with standard DFO infusions or with inadequate response to DFP monotherapy. DFP has a well-known long-term safety profile. Agranulocytosis is the most serious side effect associated with its use, occurring in about 1% of the patients. More common but less serious side effects are gastrointestinal symptoms, arthralgia, zinc deficiency, and fluctuating transaminase levels.