Subject(s)
Benzoates/therapeutic use , Iron Chelating Agents/therapeutic use , Triazoles/therapeutic use , beta-Thalassemia/drug therapy , Adult , Deferasirox , Female , Ferritins/blood , Heart , Humans , Liver/pathology , Magnetic Resonance Imaging/methods , Male , Middle Aged , Myocardium/pathology , Pilot Projects , Prospective Studies , Treatment OutcomeABSTRACT
Iron overload is not uncommon in sickle cell disease (SCD) and requires regular chelation therapy in several instances. The present study evaluates the effect of deferiprone in 15 adult patients with SCD (ten beta(s)/beta(0)thalassemia and five beta(s)/beta(s)) and iron overload. Deferiprone was given at a dose of 75 mg/kg daily for 12 months. The evaluation considered pre- and post-treatment values of serum ferritin, urinary iron excretion, and T2 values of liver and heart obtained by magnetic resonance imaging (MRI). Eleven patients had a liver biopsy prior to starting therapy to evaluate iron concentration (LIC). Twelve patients completed the study with satisfactory compliance. In ten of them (83.3%) the serum ferritin levels decreased significantly at the end of the trial; in eight patients (66.6%) the reduction of serum ferritin was accompanied by a significant increase of their liver T2 values. All patients had a significant increase of urinary iron excretion in response to the drug. Ferritin levels and liver T2 values correlated with liver iron concentration; on the contrary, ferritin levels and liver T2 values failed to show any correlation with heart T2 values. Heart T2 values did not also show any correlation with left ventricular ejection fraction. Deferiprone was well tolerated and did not cause any significant adverse effects. These results suggest that deferiprone may effectively decrease the iron deposition in patients with SCD; moreover, T2 MRI proves to be a reliable and rapid, noninvasive method for assessing the liver iron load in patients with SCD.
Subject(s)
Anemia, Sickle Cell/drug therapy , Iron Chelating Agents/administration & dosage , Iron Overload/drug therapy , Pyridones/administration & dosage , Administration, Oral , Adult , Aged , Anemia, Sickle Cell/complications , Anemia, Sickle Cell/metabolism , Deferiprone , Drug Evaluation , Female , Ferritins/blood , Heart/diagnostic imaging , Heart/physiopathology , Humans , Iron/urine , Iron Overload/etiology , Iron Overload/metabolism , Liver/diagnostic imaging , Liver/metabolism , Magnetic Resonance Imaging , Male , Middle Aged , Radiography , beta-Thalassemia/drug therapy , beta-Thalassemia/metabolismABSTRACT
Magnetic resonance imaging (MRI) appears to be useful for monitoring iron overload in thalassaemia. We studied 106 patients with beta-thalassaemia: 80 with thalassaemia major (TM) and 26 with thalassaemia intermedia (TI). Thirty-five patients with sickle cell disease (SCD) were also evaluated. Serum ferritin, liver and myocardial T2-relaxation time and liver iron concentration (LIC) were measured. LIC values, based on biopsies from 29 patients, showed a close inverse correlation with the respective liver T2-values, along with a strong positive correlation with ferritin levels in all patients. Heart T2-values correlated with left ventricular ejection fraction in TM and SCD, but not in TI patients. Both liver and heart T2-values were significantly lower in TM patients than those of TI, and SCD patients. Ferritin levels showed a strong correlation with liver T2-values in all three groups of patients. Similarly, a negative correlation was found between serum ferritin levels and heart T2-values in TM, but not in TI and SCD patients. Heart and liver T2-values showed a significant correlation only in TM patients. These results suggest that the MRI technique (T2 relaxation time) used in our study, is a reliable, safe and non-invasive method for the assessment of the deposition of iron in the liver; results for the heart become reliable only when there is heavy iron deposition.
