EARLY Detection of Cardiac and Hepatic Iron Overload by T2* Magnetic Resonance in Very Young Patients with Thalassemia Major in Oman

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4262-4262
Author(s):  
Surekha Tony ◽  
Shahina Daar ◽  
Mathew Zachariah ◽  
Azza Hinai ◽  
Idris Al-Jumah ◽  
...  
Keyword(s):  
Magnetic Resonance ◽  
Iron Overload ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Hepatic Iron ◽  
Cardiac Iron ◽  
Hepatic Iron Overload ◽  
T2 Mri

Abstract Abstract 4262 Background: Despite availability of iron chelation, iron-mediated cardiac toxicity remains the leading cause of death in thalassemia major patients. Although serum ferritin is widely used as a measure of iron overload, this has been challenged by recent magnetic resonance imaging (MRI) studies. Magnetic resonance using myocardial T2* is a highly sensitive, non-invasive and reproducible technique for detection of myocardial iron content. Materials and Methods: Seventy-four children are on follow-up at the Pediatric Thalassemia Day Care Center, Sultan Qaboos University Hospital, Muscat, Oman. Twenty-seven patients above the age of 7 years underwent T2* MRI procedure, and 9 of these patients had a follow-up T2* MRI at an interval of 1 year. MRI T2* was introduced at our institution in 2007 but was performed only on patients over the age of 12 years as it was thought that younger children would be unable to comply with the requirements of the MRI examination. Initially, we found that many of our patients failed to complete the procedure for T2* MRI (28.5% failure rate) mainly because of their inability to either hold their breath in expiration or due to movement during the procedure. But after training by physiotherapy we were successful in completing the procedure in children as young as 7 years, with no failures without the use of general anesthesia, as has been practiced by some centers. Results: Previous reports reveal no detectable cardiac iron in patients with thalassemia major less than 9.5 years of age. But we have detected in our patients severe and mild cardiac iron overload at the age of 7.5 years and 9.5 years respectively. At the time of the initial T2*MRI, the patient with severe cardiac iron overload was on chelation with Desferrioxamine with sub-optimal compliance, with a ferritin of 2605 ng/ml and a T2* MRI cardiac value of 9.3 ms. Repeat T2* MRI after 18 months (despite extensive counseling and optimization of chelation) revealed a cardiac T2* value of 4.8 ms at a ferritin level of 2796 ng/ml revealing that cardiac siderosis worsened despite the fairly constant ferritin level and the patient was shifted to combination chelation therapy. Also 44.5 % of our patients have moderate to severe hepatic iron overload. All these children were on regular 3–4 weekly follow-up for transfusion therapy with serial monitoring of ferritin levels guiding the chelation therapy. Of these, 62.9% (n=17) are on Deferiprone monotherapy at a mean dose 85.7 mg/kg, 33.3 % (n=9) are on combination chelation therapy with Deferiprone and Desferrioxamine, mean dose 95.6 mg/kg and 36.6 mg/kg respectively, and 14.2% (n=1) on Deferasirox at a dose of 40 mg/kg. Our results revealed inadequate iron chelation in some of our patients, most probably due to sub-optimal compliance that was not detected by serial ferritin monitoring (mean =1309 ng/ml). Moreover there was a poor correlation of ferritin to cardiac T2* and hepatic T2* values. Conclusions: With compliance to chelation therapy being a major issue in our patients, and failure of ferritin levels to predict the severity of cardiac and hepatic iron overload in some of the patients in a younger age group; we emphasize the importance of early and routine T2* MRI to detect organ iron overload for timely intervention with optimal iron chelation therapy in patients with thalassemia major. Disclosures: No relevant conflicts of interest to declare.

Association Between Cardiac Iron Clereance and Hepatic Siderosis

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4833-4833
Author(s):  
Alessia Pepe ◽  
Laura Pistoia ◽  
Domenico D'Ascola ◽  
Maria Rita Gamberini ◽  
Francesco Gagliardotto ◽  
...  
Keyword(s):  
Iron Overload ◽  
Iron Concentration ◽  
Thalassemia Major ◽  
Hepatic Iron ◽  
Liver Iron Concentration ◽  
Liver Iron ◽  
Cardiac Iron ◽  
Follow Up Study ◽  
Hepatic Iron Overload

Abstract Introduction. The aim of this multicenter study was to evaluate in thalassemia major (TM) if the cardiac efficacy of the three iron chelators in monotherapy was influenced by hepatic iron levels over a follow up of 18 months. Methods. Among the 2551 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network we evaluated prospectively the 98 patients those with an MR follow up study at 18±3 months who had been received one chelator alone between the 2 MR scans and who showed evidence of significant cardiac iron (global heart T2*<20 ms) at the basal MRI. Iron overload (IO) was measured by T2* multiecho technique. We used cardiac R2* (equal to 1000/T2*) because cardiac R2* is linearly proportional to cardiac iron and hepatic T2* values were converted into liver iron concentration (LIC) values. Results. We identified 3 groups of patients: 47 treated with deferasirox (DFX), 11 treated with deferiprone (DFP) and 40 treated with desferrioxamine (DFO). Percentage changes in cardiac R2* values correlated with changes in LIC in both DFX (R=0.469; P=0.001) and DFP (R=0.775; P=0.007) groups. All patients in these 2 groups who lowered their LIC by more than 50% improved their cardiac iron (see Figure 1). Percentage changes in cardiac R2* were linearly associated to the log of final LIC values in both DFX (R=0.437; P=0.002) and DFP groups (R=0.909; P<0.0001). Percentage changes in cardiac R2* were not predicted by initial cardiac R2* and LIC values. In each chelation group patients were divided in subgroups according to the severity of baseline hepatic iron overload (no, mild, moderate, and severe IO). The changes in cardiac R2* were comparable among subgroups (P=NS) (Figure 2). Conclusion. In patients treated with DFX and DFP percentage changes in cardiac R2* over 18 months were associated with final LIC and percentage LIC changes. In each chelation group percentage changes in cardiac R2* were no influenced by initial LIC or initial cardiac R2*. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures Pepe: Chiesi Farmaceutici and ApoPharma Inc.: Other: Alessia Pepe is the PI of the MIOT project, that receives no profit support from Chiesi Farmaceutici S.p.A. and ApoPharma Inc..

scholarly journals Evaluation of cardiac and hepatic iron overload in thalassemia major patients with T2* magnetic resonance imaging

Hematology ◽  
2017 ◽  
pp. 1-7 ◽  
Author(s):  
Pustika Amalia Wahidiyat ◽  
Felix Liauw ◽  
Damayanti Sekarsari ◽  
Siti Ayu Putriasih ◽  
Vasili Berdoukas ◽  
...  
Keyword(s):  
Magnetic Resonance Imaging ◽  
Magnetic Resonance ◽  
Iron Overload ◽  
Thalassemia Major ◽  
Hepatic Iron ◽  
Resonance Imaging ◽  
Hepatic Iron Overload

Update in Combined Chelation Therapy with Deferoxamine and Deferiprone in Brazilian Patients with Thalassemia Major: Assessment of Three Years

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 5415-5415
Author(s):  
Sandra Regina Loggetto ◽  
Mônica Veríssimo ◽  
Antônio Fabron Júnior ◽  
Giorgio Roberto Baldanzi ◽  
Nelson Hamerschlak ◽  
...  
Keyword(s):  
Adverse Events ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Chelation Therapy ◽  
Combined Therapy ◽  
Thalassemia Major ◽  
Liver Iron ◽  
Cardiac Iron ◽  
Cardiac Iron Overload

Abstract Introduction: Cardiac failure is a main cause of morbidity and mortality in patients with thalassemia major (TM) who are receiving regular blood transfusion due to iron overload. So, effective and adequate iron chelation is extremely important. Deferoxamine (DFO), the most widely used iron chelator, has poor compliance. Combined therapy with Deferiprone (DFP) increases chelation efficacy, decreases iron-induced complications, improves compliance increasing survival in thalassemia. Objectives: Assessment of efficacy and safety in combined chelation with DFP and DFO in thalassemic patients with iron overload. Methods and results: We have 50 thalassemia major patients in 4 Brazilian Centers (Boldrini Hospital, Sao Paulo Hematology Center, HEMEPAR and FAMEMA) receiving combined chelation therapy with follow up to three years. DFP (75–100 mg/kg/daily) and DFO (30–60 mg/kg, 4–7 days/week) are being administered during one to three years. Median age of this group is 21,5 y/o (range 8–35), with 48% female. Median age to start regular transfusions was 12 months (range 2–140) and to begin chelation therapy was 57 months (range 17–216). All patients were screened for Hepatitis C and 26% had positive sorology and/or PCR. Statistical analysis were made with Spearman test and Fisher test. All patients, except two, did cardiac and liver MRI in the initial phase of the study, resulting in 60,5% with cardiac iron overload (T2*&lt;20ms), being severe in 31,2%. Assessment of liver iron concentration (LIC) showed 95,7% with liver iron overload (&gt;3ug/g dry weight), being severe in 17,4%. During follow up, only 43 patients (86%) was screened with MRI. From these, 67,4% had cardiac iron overload (severe in 32,5%) and 78,6% had liver iron overload (severe in 11,9%). Mean serum ferritin before and after three years were 3095,7 ±1934,5 ng/ml and 2373,9±1987,6 ng/ml, respectively. Our data showed positive correlation between serum ferritin, LIC and ALT, even in initial data and after combined chelation therapy (p&lt;0,001), but there is no correlation between cardiac T2* and LIC and between cardiac T2* and ferritin. DFP adverse events included 8% agranulocytosis, 22% neutropenia, 20% arthralgia and 38% gastric intolerance. DFO adverse events were 2,6% deafness, 2,0% cataract and 12% growth deficit. Hepatic toxicity was found in 6%, but without necessity to stop treatment. Compliance in this group was excellent in 48%, good in 22% and poor in 30%. Conclusions: This is the first multicenter study to evaluate combined chelation therapy in Brazil based on cardiac MRI and LIC. Most patients had cardiac and hepatic iron overload probably because they began iron chelation lately, due to difficult access to iron chelators in the past. Cardiac iron overload didn’t have correlation with ferritin and LIC and these data need more understanding. Age of initial regular blood transfusion, increased transfusional requirement, inadequate chelation or delayed chelation may play a role in this question. Combined therapy with DFO and DFP is effective to decrease serum ferritin and LIC. Follow up and improving compliance may decrease cardiac iron overload. Adverse events are similar to literature. Combined therapy is safety in TM patients with transfusional iron overload.

scholarly journals Prospective Changes of Cardiac and Hepatic Iron and Cardiac Function in Low and Intermediate-1 Risk MDS Patients

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1911-1911
Author(s):  
Alessia Pepe ◽  
Antonella Meloni ◽  
Giancarlo Carulli ◽  
Esther Natalie Oliva ◽  
Francesco Arcioni ◽  
...  
Keyword(s):  
Cardiac Function ◽  
Iron Overload ◽  
Myocardial Fibrosis ◽  
Chelation Therapy ◽  
Mean Difference ◽  
Volume Index ◽  
Hepatic Iron ◽  
Cardiac Iron ◽  
Biventricular Function ◽  
Hepatic Iron Overload

Abstract Background: In patients with myelodysplastic syndrome (MDS) no longitudinal studies on myocardial and hepatic iron and on cardiac function and fibrosis are available in literature. So, the aim of our study was to assess the changes in cardiac and hepatic iron overload and the morpho-functional cardiac parameters by Magnetic Resonance Imaging (MRI) in MDS patients enrolled in the MIOMED (Myocardial Iron Overload in MyElodysplastic Diseases) study who performed the follow-up (FU) MRI at 12 months. Methods: MIOMED is an observational, MRI multicentre study in low and intermediate-1 risk MDS patients who have not received regular iron chelation therapy. Out of the 51 MDS patients enrolled, 48 underwent the baseline MRI exam and 28 performed the MRI FU. This analysis was limited to patients who performed both the MRIs. Mean age was 72.8±7.6 years and 8 patients were females. MIO was assessed using a multislice multiecho T2* approach. Hepatic T2* values were assessed in a homogeneous tissue area and converted into liver iron concentration (LIC). Biventricular function parameters were quantified by cine sequences. Myocardial fibrosis was evaluated by late gadolinium enhancement (LGE) acquisitions. Results: The FU MRI was not performed for the following reasons: 4 deaths and 16 patient refusal. At baseline only one patient showed cardiac iron overload (global heart T2*=14.8 ms) but he recovered at the FU (global heart T2*=28.8 ms). He was not transfused. Out of the 27 patients without significant cardiac iron at the baseline, 26 maintained the same status at the FU while one showed cardiac iron (global heart T2*=12.3 ms). Thirteen patients (8 transfusion-dependent - TD) had hepatic iron overload (MRI LIC≥3mg/g/dw) at the baseline.For this subgroup, the baseline and the FU LIC values were, respectively, 14.9±12.0 mg/g/dw and 20.1±16.1 mg/g/dw. The increase in MRI LIC values was not significant (P=0.196). Out of the 11 patients with a baseline MRI LIC<3 mg/g/dw, two (13.3%) showed hepatic iron at the FU. Only one patient was TD but both patients had not received any chelation therapy. Serum ferritin levels were comparable at both the MRIs (923±618 vs 1168±1004 ng/ml; P=0.150). Due mainly to technical reasons, biventricular function was assesses at both baseline and FU MRIs in 22 patients. At baseline 6 patients showed a reduced left ventricular ejection fraction (LVEF) and 4 of them recovered at the FU. All patients had a baseline global heart T2*>20 ms (one with 2 segmental T2* values<20 ms). At baseline 5 patients showed a reduced right ventricular EF (RV EF) and all recovered at the FU. One patient with normal LV EF at baseline showed pathological LV EF at the and 2 patients with normal RV EF at baseline showed reduced RV EF at the FU (one patient suffered from pulmonary hypertension). At the FU we detected a significant increase in the LV end-diastolic volume index (EDVI) (mean difference: 6.5±11.3 ml/m2; P=0.015) as well as in the RV EDVI (mean difference: 7.8±9.3 ml/m2; P=0.002). The change in the LV mass index between the 2 MRIs was not significant. For 18 patients the presence of myocardial fibrosis was investigated at both baseline and FU MRIs, and this subgroup was considered. Eight patients had myocardial fibrosis at the baseline. One patient showed a subendocardial ischemic pattern and seven patients showed a non-ischemic pattern and myocardial fibrosis was detected for all of them also at the FU. At the FU one new occurrence of non-ischemic myocardial fibrosis was detected. Conclusion. The new occurrences of cardiac iron, reduced cardiac function, increased LV and RV EDVI and myocardial fibrosis and the worsening in MRI LIC values suggest the need of performing periodic MRI scans, in order to better manage these patients. Figure 1 Figure 1. Figure 2 Figure 2. Figure 3 Figure 3. Disclosures Oliva: Celgene: Consultancy, Honoraria; Novartis: Consultancy, Speakers Bureau.

Assessment of the Relationship Between Iron Overload Based on Cardiac T2* MRI and Fragmented QRS in Beta-Thalassemia Major Patients

2020 ◽  
Vol 21 (8) ◽  
Author(s):  
Yazdan Ghandi ◽  
Danial Habibi ◽  
Aziz Eghbali
Keyword(s):  
Iron Overload ◽  
Chelation Therapy ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Cardiac Iron ◽  
Fragmented Qrs ◽  
Beta Thalassemia Major ◽  
Cardiac Iron Overload ◽  
The Mean ◽  
T2 Mri

Background: Cardiac involvement in beta-thalassemia major patients is an important cause of mortality. Therefore, in these patients, timely diagnosis of cardiac disorder is essential. Objectives: The present study aimed at determining the association between cardiac iron overload and fragmented QRS (fQRS). Methods: This cross-sectional study was conducted on 40 β-TM patients, aged 5 - 40 years. The presence of fQRS was evaluated in 12-lead surface electrocardiograms. Cardiac T2* MRI was performed to determine the iron overload. The patients were divided into four groups of chelation therapy. Results: The mean age of patients was reported to be 22.50 ± 6.75 years. The groups showed no significant difference regarding gender, age, or left ventricular ejection fraction. The presence of fQRS was detected in 10 patients (25%), while T2* value was lower than 20 ms in 10 patients (25%). The mean age of patients with and without fQRS was 26.23 ± 2.71 and 19.40 ± 2.61 years, respectively (P = 0.001). The univariate analysis indicated that fQRS had a significant relationship with cardiac iron overload (OR = 5; 95% CI: 1.04 - 23.99; P < 0.044). The multiple logistic regression analysis represented a significant association between iron overload and fQRS (OR = 5.556; 95% CI: 1.027 - 30.049). The sensitivity and specificity of the fQRS against MRI were equal to 50% and 83.3% respectively. Conclusions: The absence of fQRS on ECGs could be a good predictor of the lack of cardiac iron overload in β-TM patients. The results showed that fQRS might indicate the no need for close monitoring for cardiac overload with cardiac MRI and aggressive chelation therapy.

scholarly journals Prospective Changes of Cardiac and Hepatic Iron and Cardiac Function By MR in Pediatric Thalassemia Major Patients Treated with Different Chelators or Not Chelated

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4901-4901
Author(s):  
Antonella Meloni ◽  
Lorella Pitrolo ◽  
Blandina Pagano ◽  
Giancarlo Izzi ◽  
Augusto Scaccetti ◽  
...  
Keyword(s):  
Cardiac Function ◽  
Iron Overload ◽  
Prospective Studies ◽  
Thalassemia Major ◽  
Left Ventricular ◽  
Left Ventricular Ejection ◽  
Iron Chelators ◽  
Hepatic Iron ◽  
Cardiac Iron

Abstract Background: There are no prospective studies comparing the effectiveness of the three iron chelators commercially available in preventing or decreasing iron overload in the heart and liver in pediatric thalassemia major (TM) patients. Our aim was to evaluate the changes in cardiac and hepatic iron and in cardiac function by quantitative magnetic resonance imaging (MRI) over a follow-up (FU) of 18 months in pediatric TM patients treated with one of the 3 available iron chelators in monotherapy or non chelated. Methods: Among the first 1611 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, we considered pediatric patients who had maintained the same chelation regimen between the two MRI scans. Iron overload was quantified by multiecho T2* sequence. Hepatic T2* values were converted into liver iron concentration (LIC) values. Biventricular function parameters were evaluated by cine images. Due to the low sample size, no inter-treatment comparisons were performed and intra-treatment comparison was performed only in the DFX group. Results: Four groups of patients were identified: 6 patients (3 F, 10.0±2.2 years) treated with desferioxamine (DFO– mean dosage 43.7±6.8 mg/kg/die), 7 patients (3 F, 15.5±1.7 yrs) treated with deferiprone (DFP– mean dosage 75.0±9.2 mg/kg/die), 39 patients (13 F, 13.58±3.39 yrs) treated with deferasirox (DFX– mean dosage 26.6±6.7 mg/kg/die), and 2 patients (2 F, 11.1±5.3 yrs) not chelated because they had performed a bone marrow transplantation. Compliance to chelation therapy was excellent/good in all treated groups. At baseline in DFO, DFP and no chelated groups no patient showed a global heart T2* value<20 ms. In all 4 groups all patients who showed no cardiac iron overload at baseline maintained at the FU the same status. At baseline in DFX group 6 patients (17.6%) had heart T2* values<20 ms. The 4 patients with intermediate cardiac iron (T2* 10-20 ms) at the baseline showed no iron at the FU while the patient with severe cardiac iron (T2*<10 ms) remained in the same status at the FU. Non chelated patients had higher global heart T2* values at baseline (non-chelated 37.7±0.5 ms > DFP 35.3±4.9 ms > DFX 32.7±9.6 ms > DFO 31.9±10.5 ms) while DFP patients had higher global heart T2* values at FU (DFP 39.5±6.1 ms > DFX 34.2±7.3 ms > DFO 33.6±7.9 ms > non-chelated 28.9±4.0 ms ). In the DFO group at baseline 1 patient showed pathological left ventricular ejection fraction (LVEF) and he recovered at the follow up. In the DFP group at baseline 2 patients showed pathological LVEF and both recovered at the follow up. In the DFX group at baseline 3 patients showed pathological LVEF: 2 recovered at the FU and 1 did not perform the evaluation of the cardiac function at FU due to technical reasons. Conversely 9 patients with normal LVEF at baseline showed pathological LVEF at the FU. In the DFO group the percentage of patients with MRI LIC>3 mg/g/dw went up from 83% to 100%. In the DFP group all patients showed MRI LIC>3 mg/g/dw at baseline and they maintained this status at the FU. In the DFX group the percentage of patients with MRI LIC≥3 mg/g/dw went down from 71.1% to 52.6%. The MRI LIC mean difference was -1.6±4.4 mg/g/dw (P=0.006). Only one of the two non chelated patients had a baseline MRI LIC≥3 mg/g/dw and she remained in the same status at the FU. Conclusion: This longitudinal analysis confirms significant rate of iron overload even in very young TM population, in particular in the liver. In this population, DFP seems to be more effective in the heart with a concordant positive effect on the global systolic function. Conversely, DFX seems to be more effective in the liver. However, further prospective studies are needed on larger study population to confirm the data. Figure 1 Figure 1. Disclosures Pepe: Chiesi: Speakers Bureau; ApoPharma Inc.: Speakers Bureau; Novartis: Speakers Bureau.

Magnetic Resonance Imaging (MRI) Detection of Iron Overload In Patients with Myelodysplastic Syndrome (MDS)

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4960-4960
Author(s):  
Fabio PS Santos ◽  
Claudia Bley ◽  
Ricardo Helman ◽  
Guilherme Fleury Perini ◽  
Iracema Esteves ◽  
...  
Keyword(s):  
Iron Overload ◽  
Transferrin Saturation ◽  
Low Risk ◽  
Refractory Anemia ◽  
Categorical Variables ◽  
Hepatic Iron ◽  
Cardiac Iron ◽  
Hepatic Iron Overload ◽  
Cardiac Iron Overload ◽  
T2 Mri

Abstract Abstract 4960 Introduction: Transfusion dependent anemia and iron overload are associated with reduced survival in patients with MDS. Increased iron absorption at the gastrointestinal tract may also contribute to iron overload. Serum ferritin is the most common method of assessing body iron content, but it can be elevated in patients with inflammatory conditions, and may not correlate with iron overload in specific organs such as the heart. T2* MRI is a non-invasive method for detecting iron overload in patients with transfusion-dependent anemia, and its efficacy has been validated in patients with thalassemia major. There are few studies reporting on the efficacy of T2* MRI for detection of iron overload in patients with MDS. Objective: To evaluate the efficacy of T2* MRI in detection of iron overload in patients with MDS, the prevalence of iron overload in this disease and correlate MRI findings with iron indexes (ferritin, transferrin and non-transferrin bound iron [NTBI]). Methods: Patients with MDS or chronic myelomonocytic leukemia (CMML), independent of transfusion requirements, were recruited into a prospective, single center trial to assess the efficacy of T2* MRI for detection of iron overload in this scenario. Patients receiving iron chelation therapy were excluded. Iron indexes were measured at the time of T2* MRI evaluation. Hepatic iron overload was considered in patients with a hepatic iron concentration (HIC) ≥ 2 g/mg. Cardiac iron overload was considered in patients with a T2* value < 20 milliseconds. Mann-Whitney and Fischer exact tests were used to compare baseline continuous and categorical variables among patients with and without iron overload as assessed by HIC. Correlation between HIC and iron indexes was assessed with Spearman correlation. Results: A total of 37 patients with MDS and one patient with CMML were recruited. Three patients were not evaluated by MRI due to claustrophoby, so 35 patients remain for the analysis. Median age was 68 years (range 18–84). MDS subtypes by the WHO classification include refractory anemia (N=3), refractory anemia with ring sideroblasts (N=5), 5q- syndrome (N=3), refractory cytopenias with multilineage dysplasia (N=13), refractory anemia with excess blasts-I (N=6) and –II (N=3) and unclassifiable MDS (N=1). Information about transfusion requirement was available for 28 patients, and 14 (50%) were transfusion dependent. Twenty-two patients could be classified by the WHO Prognostic Score System (WPSS) and were categorized as very low-risk (N=6), low-risk (N=3), intermediate risk (N=6) and high risk (N=7). Median ferritin, transferrin saturation and NTBI values were 1079.6 ng/mL (range 21.8–12738 ng/mL), 63% (range 6–100%) and 0.34 microM (range 0–12.93 microM), respectively. Median cardiac T2* value was 45.3 ms (range 19.7–70.1 ms), and only one patient had a T2* value indicative of cardiac iron overload. Median HIC value was 3.31 g/mg (range 0.2–9.97 g/mg), and 66% of patients had hepatic iron overload. Patients with hepatic iron overload had higher ferritin levels (1181 ng/mL vs. 131 ng/mL, p=0.007) and transferrin saturation (64% vs. 39%, p=0.02), but no differences in NTBI (0.29 microM vs. 0.22 microM, p=0.42). Patients with elevated HIC had a higher prevalence of transfusion dependency but the difference was not significant (50% vs. 33%, p=0.67). Ferritin levels and transferrin saturation correlated with HIC (r = 0.552, p=0.001 [ferritin]; r = 0.609, p=0.001 [transferrin saturation]). Conclusion: T2* MRI can detect iron overload in patients with MDS. Iron overload in MDS cannot be solely explained by transfusion dependent anemia. The study is currently ongoing and updated results will be presented at the meeting. Disclosures: No relevant conflicts of interest to declare.

Combination Therapy of Deferasirox and Deferoxamine As Paregoric Treatment of Severe Iron Overload in Patients with Thalassemia Major

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 5181-5181
Author(s):  
Vassilis Ladis ◽  
Dimitra Kyriacopoulou ◽  
Konstantinos Stokidis ◽  
Aggeliki Moira ◽  
Antonios Kattamis
Keyword(s):  
Combination Therapy ◽  
Iron Overload ◽  
Research Funding ◽  
Chelation Therapy ◽  
Combined Treatment ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Duration Of Treatment ◽  
Considerable Problem

Abstract Abstract 5181 Background: Despite the recent availability of multiple therapeutic options for chelation therapy, severe iron overload remains a significant cause of morbidity and mortality in a small group of patients with thalassemia major (TM). Every effort to induce negative iron balance for these patients is warranted. In this context, combination of deferasirox (DFX) and deferoxamine (DFO) may be of significant value. In this report, we present the paregoric use of DFX and DFO in severely iron overloaded thalassemic patients. Patients and Methods: Five patients (3 Males, 2 females) have been treated with DFX and DFO as a last rescue measure. In these patients previous combined treatment with DFO and DFP had failed. Informed consent and approval for the use of this schema as paregoric therapy was obtained by patients and hospital. The main inclusion criteria for this treatment were: LIC>30 mg/gr d. w. and T2* cardiac <10 msec. Treatment consisted of daily DFX at 30–40 mg/kg/day and DFO at 40–50 mg/kg/day for 3–6 days/week. Results: Patients' characteristics, results and toxicity are shown in table 1 and 2. Improvement in iron load status ranged widely during a follow-up from 1 to 3 yrs. LIC reduced in 4/5 patients, cardiac T2* increased in 3/5 patients, while LVEF showed no significant changes. Ferritin levels improved in 2/5 patients. Deterioration of safety parameters necessitated not to discontinue treatment. Conclusions: These data in the use of combination therapy of DFX and DFO suggest a potential modality of chelation therapy in severely iron overloaded patients. Response was variable and seemed not to be always related to compliance or the duration of treatment. Increase of serum creatinin and AST in all patients is a considerable problem in patients with persistent significant iron overload and already impaired hepatic and renal function, despite having been treated with different schema of iron chelation. Longer and cautious follow-up is needed to come to reliable conclusions. Disclosures: Ladis: Novartis: Consultancy, Honoraria, Research Funding; Apopharma: Consultancy, Honoraria, Research Funding. Kattamis:Apopharma: Honoraria, Speakers Bureau; Novartis: Honoraria, Research Funding, Speakers Bureau.

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