Longitudinal follow‐up of patients with thalassaemia intermedia who started transfusion therapy in adulthood: a cohort study

Abstract

We longitudinally evaluated the effects of regular blood transfusions (BTs), in the real-life context of the Myocardial Iron Overload in Thalassaemia network, in patients with thalassaemia intermedia (TI). We considered 88 patients with TI (52 females) who started regular BTs after the age of 18 years. Magnetic resonance imaging was used to quantify iron overload and biventricular function. For 56Á8% of the patients there were more than two indications for the transition to regular BTs, with anaemia present in 94Á0% of the cases. A significant decrease in nucleated red blood cells, platelets, lactate dehydrogenase, bilirubin, and uric acid levels was detected 6 months after starting regular BTs. After the transition to the regular BT regimen there was a significant increase only in the frequency of hypothyroidism and osteopenia, and a significant decrease in liver iron and cardiac index. The percentage of chelated patients increased significantly after starting regular BTs. The decision to regularly transfuse patients with TI may represent a way to prevent or slow down the natural progression of the disease, despite the more complex initial management.

Survival and complications in patients with haemoglobin E thalassaemia in Sri Lanka: a prospective, longitudinal cohort study

“… 18 , 19 Another unanticipated finding was the striking subacute development of intolerance to anaemia in older patients (aged 27–39 years) who had previously thrived without regular transfusions, consistent with our previous studies of age-related changes in adaptation to severe anaemia, 20 and similar to findings of a previous study of older patients with thalassaemia intermedia not involving haemoglobin E, all of whom had been started on scheduled transfusions after age 18 years, and were in a similar age range to our patients. 21 We hypothesise that this finding might be related to changes in responsiveness to erythropoietin with age, as previously observed; 20 measurements of serum erythropoietin are awaited to provide secure conclusions. Further findings will be published subsequently.…”

A complication risk score to evaluate clinical severity of thalassaemia syndromes

“…Although recognised as the most severe form, TDT has witnessed improvement in survival, especially in Western countries, owing to advances in chelation treatment and transfusion safety. Combined, these observations indicate that the currently recognised clinical forms of TS have varying levels of similarity in clinical severity, which makes clear distinction and management decisions essential to prevent or slow down the natural progression of the disease, despite the more complex initial management 11 …”

Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

“… 6 Improvement in hemolysis markers, nucleated red cells and cardiac index have also been reported in longitudinal studies of NTDT patients who were started on regular transfusions in adulthood. 10 This also explains why iron chelation did not seem to have a role in preventing cardiovascular deaths in this cohort. Thus, a trial of chronic transfusion in patients at risk of significant morbidity may be justified but this needs to be weighed against the eventual risk of secondary siderosis and the elevated need and high cost of iron chelation therapy in a regular transfusion setting.…”