Title |
Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone
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Published in |
Critical Reviews in Diagnostic Imaging, May 2013
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DOI | 10.1186/1532-429x-15-38 |
Pubmed ID | |
Authors |
John B Porter, John Wood, Nancy Olivieri, Elliott P Vichinsky, Ali Taher, Ellis Neufeld, Patricia Giardina, Alexis Thompson, Blaine Moore, Patricia Evans, Hae-Young Kim, Eric A Macklin, Felicia Trachtenberg |
Abstract |
BACKGROUND: Established heart failure in thalassaemia major has a poor prognosis and optimal management remains unclear. METHODS: A 1 year prospective study comparing deferoxamine (DFO) monotherapy or when combined with deferiprone (DFP) for patients with left ventricular ejection fraction (LVEF) <56% was conducted by the Thalassemia Clinical Research Network (TCRN). All patients received DFO at 50--60 mg/kg 12--24 hr/day sc or iv 7 times weekly, combined with either DFP 75 at mg/kg/day (combination arm) or placebo (DFO monotherapy arm). The primary endpoint was the change in LVEF by CMR. RESULTS: Improvement in LVEF was significant in both study arms at 6 and 12 months (p = 0.04), normalizing ventricular function in 9/16 evaluable patients. With combination therapy, the LVEF increased from 49.9% to 55.2% (+5.3% p = 0.04; n = 10) at 6 months and to 58.3% at 12 months (+8.4% p = 0.04; n = 7). With DFO monotherapy, the LVEF increased from 52.8% to 55.7% (+2.9% p = 0.04; n = 6) at 6 months and to 56.9% at 12 months (+4.1% p = 0.04; n = 4). The LVEF trend did not reach statistical difference between study arms (p = 0.89). In 2 patients on DFO monotherapy during the study and in 1 patient on combined therapy during follow up, heart failure deteriorated fatally. The study was originally powered for 86 participants to determine a 5% difference in LVEF improvement between treatments. The study was prematurely terminated due to slow recruitment and with the achieved sample size of 20 patients there was 80% power to detect an 8.6% difference in EF, which was not demonstrated. Myocardial T2* improved in both arms (combination +1.9 +/- 1.6 ms p = 0.04; and DFO monotherapy +1.9 +/- 1.4 ms p = 0.04), but with no significant difference between treatments (p = 0.65). Liver iron (p = 0.03) and ferritin (p < 0.001) both decreased significantly in only the combination group. CONCLUSIONS: Both treatments significantly improved LVEF and myocardial T2*. Although this is the largest and only randomized study in patients with LV decompensation, further prospective evaluation is needed to identify optimal chelation management in these high-risk patients. |
X Demographics
Geographical breakdown
Country | Count | As % |
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United States | 1 | 25% |
Unknown | 3 | 75% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 4 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 67 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Master | 11 | 16% |
Student > Ph. D. Student | 7 | 10% |
Student > Doctoral Student | 6 | 9% |
Student > Bachelor | 6 | 9% |
Other | 6 | 9% |
Other | 16 | 24% |
Unknown | 15 | 22% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 40 | 60% |
Nursing and Health Professions | 3 | 4% |
Neuroscience | 2 | 3% |
Psychology | 2 | 3% |
Agricultural and Biological Sciences | 1 | 1% |
Other | 3 | 4% |
Unknown | 16 | 24% |