Pf789 8‐years’ Overall and Event Free Survival Is Suboptimal in a Large Group of Transfusion‐dependent Thalassemic Patients

Abstract

Background:In 2005, a national registry was established to collect epidemiological and clinical data for patients with beta-thalassemia living in France. The objective of this data collection was to better understand patient characteristics, treatment patterns, and clinical outcomes among patients followed in France. Completeness is achieved by cross-checking with different sources. The registry collects data on patients with beta-thalassemia major TM) (defined as requiring ≥8 RBC transfusions per year) and thalassemia intermedia (TI). Data is updated every 2 years. Aims: Here, we first describe the whole population included in the registry with the number and causes of death during the period 2005-2017; second, we focus on a sub-group of transfusion-dependent thalassemia (TDT) patients followed from 2009/2010 for 2 to 6 years. Methods: Patients were included between 01/01/2005 and 31/01/2019. A cross sectional study and a longitudinal study were performed in February 2019. For the longitudinal study, TDT (either TM or TI) patients included in the registry before 2010 were selected. Baseline data corresponds to data collected in 2009 or 2010 and 2-, 4-, and 6-year data corresponds to data collected during follow-up. Iron overload was assessed by serum ferritin (SF) level and hepatic and cardiac MRI. Iron measurement risk category cutoffs based on published literature were ≤20 ms (intermediate risk) and ≤10 ms (high risk) for cardiac T2 * and ≥7

The Outcomes of Patients with Haemoglobin Disorders in Cyprus: A Joined Report of the Thalassaemia International Federation and the Nicosia and Paphos Thalassaemia Centres (State Health Services Organisation)

“…The most frequent complications were bone disease, mainly osteoporosis, followed by hypogonadism, diabetes, hypothyroidism, and heart disease. A comparison of complication rates with a Greek β-thalassaemia cohort [16] is provided in Supplementary Table S1.…”