The results of this study have the potential to affect the nearly 100,000 people born worldwide with this severe blood disorder every year. Frequent blood transfusions, necessary for treatment of thalassemia major, unfortunately carry the adverse side effect of iron build-up in the body, which can damage the heart. In fact, cardiac disease is responsible for 70 percent of deaths in thalassemia major patients.
A team of researchers from University College London Hospitals studied 81 thalassemia major patients who had no previous cardiac history and whose LVEF readings were monitored on a yearly basis for a median of six years to a maximum of 20 years, depending on the patient.
A normal LVEF reading was 55 percent by the methodology used in this study. It was determined that if an LVEF reading was less than 45 percent (indicating a weakening of the heart muscle), or if the reading fell 10 percent between consecutive measurements, the patient's risk of developing symptomatic heart disease was 25 times greater than in patients with normal readings.
Thirty-four patients in the study group were found to have this risk factor, and, in these cases, intense treatment with deferoxamine mesylate (DFO), a drug given intravenously or by injection, was recommended for removing the excess iron from the patient's blood. After rapid improvement in heart function with the initial treatment, the drug continued to make more gradual improvements over several years of sustained treatment before achieving its maximum effect.
All 27 patients who complied with the recommended therapy survived, while the seven who did not comply with treatment died from cardiac disease. The study found that the median period of time between an abnormal LVEF reading and development of cardiac disease was three and a half years – enough time for treatment with DFO to prevent and reverse the severe heart dysfunction.
According to Professor John B. Porter of the Royal Free and University College Medical School, the senior author of the study, "This study shows that regular monitoring of LVEF, combined with sustained DFO treatment when necessary, can be used to achieve excellent long-term prognosis in patients with thalassemia major and should be an integral part of their medical care."
Sonali M. Smith, M.D., Assistant Professor in the Section of Hematology/Oncology at the University of Chicago's Department of Medicine, notes, "The most promising aspect of the study is that the combination of careful monitoring, prompt institution of treatment before symptoms appear, and good patient compliance improves survival. This study not only supports routine monitoring, but provides strong evidence that prompt intervention based on changes in heart function is effective. The impact of the study is that it will influence practice patterns and provide direct patient benefit."
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To receive a copy of the study or to arrange an interview with Professor John B. Porter, senior author of the study, please contact Aislinn Raedy at 202-776-0544 or araedy@hematology.org.
Members of the media are invited to attend the 46th ASH Annual Meeting and Exposition, scheduled for December 4-7, 2004, in San Diego, Calif. For more information, please visit www.hematology.org/news/.
The American Society of Hematology (www.hematology.org) is the world's largest professional society concerned with the causes and treatment of blood disorders. Its mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems, by promoting research, clinical care, education, training, and advocacy in hematology.
Blood, the official journal of the American Society of Hematology, is the most cited peer-reviewed publication in the field. Blood is issued to Society members and other subscribers twice per month, available in print and online at www.bloodjournal.org.
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