PF789 8‐YEARS’ OVERALL AND EVENT FREE SURVIVAL IS SUBOPTIMAL IN A LARGE GROUP OF TRANSFUSION‐DEPENDENT THALASSEMIC PATIENTS

Background: Current therapeutic modalities with proper transfusion regimen and chelation therapy have dramatically increased life expectancy and decreased anemia and iron overload related complications in transfusion dependent thalassemic (TDT) patients. In many countries where thalassemia prevention programs were introduced, the majority of TDT population is of middle age. Data on morbidity and new complications on this ageing thalassemics are sparse. Aims: To determine the 8‐years’ overall survival (OS) and event‐free survival (EFS) and to examine factors affecting survival in a large group of TDT patients. Methods: This is a retrospective analysis of mortality and morbidity in a large group of homogenously –treated TDT patients in a single institution. Patients with TDT, regularly transfused in our unit on 31/12/2010, were followed for 8 years till 31/12/2018. ‘New event’ was defined as a new complication significant enough to require therapeutic intervention. The effect of different factors (like age, splenectomy, history of hepatitis C, burden of transfusions, iron overload, etc) on mortality and morbidity was evaluated. Burden of transfusion was evaluated as continuous (cc of transfused red blood cells/kg/year) and dichotomous variable (up to, and, more than 10 units/12 weeks). The majority of adult TDT patients (76.4%) received >10 units/12 weeks. Results: 435 patients (mean 33.3 ± 7.9 years, max 52.3 years) were longitudinally followed. During an observation period of 8 years, 30 deaths occurred, corresponding to an 8‐years OS of 93.1%. Deaths significantly increased after the age of 40 years (figure). Splenectomy and history of hepatitis C were risk factors for death (O.R. 7.0 and 4.5, respectively, both p  < 0.05). Improvement of iron overload was noted between 2010 and 2018 (ferritin levels: 2108.6 ± 2098 vs. 1550.8 ± 1883.5 μg/l, respectively, p  < 0.05). During the observation period, percentages of patients experiencing new events were as follows: hypothyroidism 20.7%, cardiac complications 8.6%, glucose homeostasis disturbances 7.3%, hypogonadism 21.6%, significant osteoporosis 6.4%, renal failure 1%, neurological complications 5.5% and cancer 6.1%. 8 years’ EFS was 55%. Apart from age, no other factors were related to the occurrence of new events. Summary/Conclusion: Despite adequate transfusion regimen and improvement on iron overload, OS and EFS of TDT patients remain suboptimal, with mortality increasing significantly after the 4 th decade of life. Contributing factors to death, like splenectomy and history of hepatitis C, may be related to the initial treatment period. Effect of transfusion burden needs further evaluation. Ageing TDT population is likely to develop more complications with advanced age with the occurrence of malignancies being a major concern.