Survival in a large cohort of Greek patients with transfusion- dependent beta thalassaemia and mortality ratios compared to the general population Vassilis Ladis 1 , Giorgos Chouliaras 1 , Vasilios Berdoukas 1 , Antonia Chatziliami 2 , Christina Fragodimitri 2 , Fotis Karabatsos 2 , Jacqueline Youssef 2 , Antonis Kattamis 1 , Markissia Karagiorga-Lagana 2 1 Thalassaemia Unit, 1st Department of Paediatrics, University of Athens, ‘Aghia Sophia’ Children’s Hospital, Athens; 2 Thalassaemia Unit, National Health System, ‘Aghia Sophia’ Children’s Hospital, Athens, Greece With adequate transfusion regimes and iron chelation therapy over the last 30 years, transfusion-dependent beta thalassaemia (TDT) changed from resulting in early death to being compatible with prolonged survival. Although a number of studies have shown that deaths from cardiac disease have reduced significantly (1–3), cardiac dysfunction remains the most common cause of premature death. However, to date, no evaluation of the risk of death in thalassaemia compared to the general population has been made, and it is unknown whether this relative risk is reducing over time. New chelation options have clearly shown reduced car- diac morbidity and mortality (4–6). These options include, in particular, the use of deferiprone, combina- tion therapy with deferoxamine and deferiprone and intensive deferoxamine therapy. A further advance is that based on magnetic resonance imaging (MRI) findings, physicians can now tailor the appropriate chelation regime according to their concerns with respect to iron loading. Recently, MRI has been demonstrated not only to measure the total body iron but also to allow organ- specific iron assessments, particularly the heart (7–11). In addition, advances in the management of hepatitis C, early diagnosis and management of diabetes and other endocrinopathies, and careful attention to the possibility of serious life-threatening infections are also important components of patients’ general well-being and extended survival. In this work, based on a very large number of uni- formly followed and treated patients, we present the results on survival as well as the causes of death in trans- fusion-dependent beta thalassaemia in the two largest Abstract Background: With transfusions and chelation therapy, the prognosis for transfusion-dependent beta thalas- saemia has changed from being fatal in early childhood to a chronic disorder with prolonged sur- vival. Design and methods: In this historical prospective study, we present survival, causes of death and mortality ratios compared to the general population in 1044 Greek patients with transfusion-dependent beta thalassaemia. Results: At the age of 50 years, the overall survival was 65.0%, while the cardiac death–free survival was 77%. Birth cohort had a significant effect on survival (P < 0.001) with a negative trend towards past decades. The standardised mortality ratio (standardised for sex and ages 20–40 years) compared to the general population improved significantly from 28.9 in 1990–1999 to 13.5 in 2000–2008, while the standardised cardiac mortality ratio reduced from 322.9 to 106.6, respectively. Conclusions: Survival in thalassaemia has dramatically improved over the last twenty years but mortality remains significantly increased, compared to the general population. Keywords transfusion-dependent beta thalassaemia; survival; cardiac death; iron overload; standardised mortality ratio Correspondence Giorgos Chouliaras, MD, Thalassaemia Unit, 1st Department of Paediatrics, University of Athens, ‘Aghia Sophia’ Children’s Hospital, Thivon & Papadiamantopoulou str, 11527, Athens, Greece and Evrou 89, 11527, Athens, Greece. Tel: 00302107467772; Fax: 00302107467772; e-mails: georgehouliaras@msn.com, gchoul@med.uoa.gr Accepted for publication 31 January 2011 doi:10.1111/j.1600-0609.2011.01582.x ORIGINAL ARTICLE European Journal of Haematology 86 (332–338) 332 ª 2011 John Wiley & Sons A/S