7 research outputs found
Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload
<p>Abstract</p> <p>Background</p> <p>Thalassaemia major (TM) patients need regular blood transfusions that lead to accumulation of iron and death from heart failure. Deferiprone has been reported to be superior to deferoxamine for the removal of cardiac iron and improvement in left ventricular (LV) function but little is known of their relative effects on the right ventricle (RV), which is being increasingly recognised as an important prognostic factor in cardiomyopathy. Therefore data from a prospective randomised controlled trial (RCT) comparing these chelators was retrospectively analysed to assess the RV responses to these drugs.</p> <p>Methods</p> <p>In the RCT, 61 TM patients were randomised to receive either deferiprone or deferoxamine monotherapy, and CMR scans for T2* and cardiac function were obtained. Data were re-analysed for RV volumes and function at baseline, and after 6 and 12 months of treatment.</p> <p>Results</p> <p>From baseline to 12 months, deferiprone reduced RV end systolic volume (ESV) from 37.7 to 34.2 mL (p = 0.008), whilst RV ejection fraction (EF) increased from 69.6 to 72.2% (p = 0.001). This was associated with a 27% increase in T2* (p < 0.001) and 3.1% increase in LVEF (p < 0.001). By contrast, deferoxamine showed no change in RVESV (38.1 to 39.1 mL, p = 0.38), or RVEF (70.0 to 69.9%, p = 0.93) whereas the T2* increased by 13% (p < 0.001), but with no change in LVEF (0.32%; p = 0.66). Analysis of between drugs treatment effects, showed significant improvements favouring deferiprone with a mean effect on RVESV of -1.82 mL (p = 0.014) and 1.16% for RVEF (p = 0.009). Using regression analysis the improvement in RVEF at 12 months was shown to be greater in patients with lower baseline EF values (p < 0.001), with a significant difference in RVEF of 3.5% favouring deferiprone over deferoxamine (p = 0.012).</p> <p>Conclusion</p> <p>In this retrospective analysis of a prospective RCT, deferiprone monotherapy was superior to deferoxamine for improvement in RVEF and end-systolic volume. This improvement in the RV volumes and function may contribute to the improved cardiac outcomes seen with deferiprone.</p
IRON OVERLOAD, CARDIAC AND OTHER FACTORS AFFECTING PREGNANCY IN THALASSEMIA MAJOR
The reproductive thalassemic population is growing older and doctors
confront the challenge of the thalassemic pregnancy. Pregnancy is
characterized by dynamic multiple system changes, resulting in increased
basal oxygen consumption, changes in energy substrate use by different
organs and increased susceptibility to oxidative stress, while
homozygous transfusion-dependent beta-thalassemia (beta-thal) patients
manifest cardiac, hepatic, endocrine, and metabolic disorders
attributable to chronic anoxia and iron overload. Pregnant thalassemic
patients require significantly larger amount of total blood transfusion
during pregnancy and iron overload increases the oxidative stress of
pregnancy, while the risk for cardiovascular events, in a high cardiac
output state, is augmented and chelation treatment is generally avoided
due to the potential teratogenicity. Pregnancy in thalassemia major
should be considered high risk, and be cared for by an expert team with
special caution and sensitivity
Predictive echo-Doppler indices of left ventricular impairment in B-thalassemic patients
Early detection of cardiac-function impairment by echo-Doppler indices
can assist in preventing further cardiac damage by modifying disease
progression and treatment. We analyzed our thalassemia major patients
database with 10 years cardiac follow-up. Included patients were under
constant therapy and should have an initial echo-Doppler study with
normal Shortening Fraction ( SF > 30%) and reexamination within the
last year. We identified patients who developed impaired left
ventricular (LV) function in the last Echo and we attempted to find
which measured indices could predict LV function impairment. Three
hundred fifteen of the 632 database patients were enrolled. Twelve of
them developed LV systolic dysfunction. There were no statistically
significant differences in mean age, ferritin, and pretransfusion
hemoglobin levels of the two groups. LV-systolic-dysfunction group was
presenting statistically significantly higher LF end-systolic diameter
(LVESD) index, lower SF, higher early transmitral peak flow
velocities/late transmitral peak flow velocities ( A) ratios, lower A
value. All other echocardiographic parameters did not differ
significantly. By receiver-operating characteristic analysis, we
determined systolic and diastolic indices specificity and sensitivity
for LV impairment: LVESD 97% specificity, 11% sensitivity ( cutoff
value 2.44 cm/m(2)), SF 92.1 and 33.3% ( cutoff value 33%). Regarding
diastolic indices, A index was the best criterion ( 97.7% specificity,
25% sensitivity, cutoff value <= 0.36). Low values of the diastolic
index A wave and increased values of the LVESD index were the earliest
pathological findings in patients that finally developed LV systolic
dysfunction. These parameters could be used as heart-dysfunction
predictors with relatively good sensitivity and specificity
Liver disease in adult transfusion-dependent beta-thalassaemic patients: investigating the role of iron overload and chronic HCV infection
Background Iron overload and hepatitis-C virus (HCV) infection, have
been implicated in the evolution of liver disease, in patients with
transfusion-dependent beta-thalassaemia major (BTM). However, the impact
of these factors in late stages of liver disease in adults with BTM, has
not been extensively studied. Aims To investigate serum indices of iron
overload, HCV infection and liver disease, in a cohort of 211 adult
Greek patients with BTM, in relation with the findings from liver
biopsies. Methods In this cross-sectional study, 211 patients with BTM
were enrolled and studied, in relation with HCV infection, ferritin,
transaminases, chelation treatment and antiviral treatment. Based on 109
patients biopsied, we correlated liver fibrosis, haemosiderosis and
inflammation, with serum indices and HCV status Results Among all
patients, 74.4% were anti-HCV positive (HCV+). Ferritin was positively
correlated with transaminases and negatively correlated with age, while
it was not significantly different among HCV+ and HCV patients. Among
the HCV+ patients, 55.4% reported antiviral treatment, while genotype 1
predominated. In a subfraction of 109 patients, in which liver biopsy
was performed, 89% were HCV+ and 11% HCV. Fibrosis was significantly
correlated with age (P=0.046), AST (P=0.004), ALT (P=0.044) and
inflammation (P<0.001). Advanced fibrosis was present with even minimal
haemosiderosis, independently of ferritin values or HCV history.
Conclusions These data suggest that in the late stages of liver disease
in BTM patients, iron overload may be the critical determinant, since
fibrosis is related to the minimal haemosiderosis, independently of HCV
history
Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload
Background: Thalassaemia major (TM) patients need regular blood
transfusions that lead to accumulation of iron and death from heart
failure. Deferiprone has been reported to be superior to deferoxamine
for the removal of cardiac iron and improvement in left ventricular (LV)
function but little is known of their relative effects on the right
ventricle (RV), which is being increasingly recognised as an important
prognostic factor in cardiomyopathy. Therefore data from a prospective
randomised controlled trial (RCT) comparing these chelators was
retrospectively analysed to assess the RV responses to these drugs.
Methods: In the RCT, 61 TM patients were randomised to receive either
deferiprone or deferoxamine monotherapy, and CMR scans for T2* and
cardiac function were obtained. Data were re-analysed for RV volumes and
function at baseline, and after 6 and 12 months of treatment.
Results: From baseline to 12 months, deferiprone reduced RV end systolic
volume (ESV) from 37.7 to 34.2 mL (p = 0.014), whilst RV ejection
fraction (EF) increased from 69.6 to 72.2% (p = 0.001). This was
associated with a 27% increase in T2* (p < 0.001) and 3.1% increase
in LVEF (p < 0.001). By contrast, deferoxamine showed no change in RVESV
(38.1 to 39.1 mL, p = 0.38), or RVEF (70.0 to 69.9%, p = 0.93) whereas
the T2* increased by 13% (p < 0.001), but with no change in LVEF
(0.32%; p = 0.66). Analysis of between drugs treatment effects, showed
significant improvements favouring deferiprone with a mean effect on
RVESV of-1.82 mL (p = 0.013) and 1.16% for RVEF (p = 0.008). Using
regression analysis the improvement in RVEF at 12 months was shown to be
greater in patients with lower baseline EF values (p < 0.001), with a
significant difference in RVEF of 3.5% favouring deferiprone over
deferoxamine (p = 0.012).
Conclusion: In this retrospective analysis of a prospective RCT,
deferiprone monotherapy was superior to deferoxamine for improvement in
RVEF and end-systolic volume. This improvement in the RV volumes and
function may contribute to the improved cardiac outcomes seen with
deferiprone