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  • American Society of Hematology  (7)
  • 2015-2019  (7)
  • 1
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    Significant Improvement of Survival By T2* Cardiovascular Magnetic Resonance in Thalassemia Major
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 4567-4567
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 4567-4567
    Abstract: Background: In 2004 seven Italian centers reported survival data for patients with thalassemia major (TM) and showed that heart disease due to iron overload was the most common cause of death (Borgna et al Haematologica 2004). In the same years the accurate and noninvasive assessment of cardiac siderosis was made possible in Italy by the introduction of the T2* cardiovascular magnetic resonance (CMR). Purpose: We aimed to evaluate if the deployment of T2* CMR had an impact on the mortality rate. Methods: Four centers contributed to the present study, updating the data of the enrolled patients until August 31, 2010. For the patients who died, the date of the death represented the end of the study. 577 patients (264 females and 313 males) were included. Results: One-hundred and fifty-nine (27.6%) patients died, 124 of whom (77.9%) died before the year 2000. The Table shows the comparison between dead patients and survivors. Dead patients were significantly younger and they were more frequently males. Dead patients started chelation therapy significantly later. HIV, arrhythmias and heart failure were significantly more frequent in dead patients. According to the Cox model, the following variables were identified as significant univariate prognosticators for the death: male sex (HR=1.87, 95%CI=1.34-2.60, P 〈 0.0001), HIV (HR=2.55, 95%CI=1.25-5.20, P=0.010) and heart failure (HR=8.86, 95%CI=6.37-12.31, P 〈 0.0001). MRI was not performed in 406 patients (70.4%) and no patient had been scanned before his/her death. Among the survivors, MRI was not performed in the 59% of the cases (P 〈 0.0001). The absence of an MRI scan was a significant univariate prognosticator for death (HR=43.25, 95%CI=11.32-165.33, P 〈 0.0001). The study was restricted to the patients dead after 2004 (19/159=12%) or followed until August 2010 (N=357). In this subgroup of 376 patients, MRI was not performed in the 52.4% of the survivors and in all dead patients (P 〈 0.0001). The absence of a MRI exam was reconfirmed as a strong predictive factor for death (HR=49.37, 95%CI=1.08-2263.24, P=0.046). The Kaplan-Meier curve is showed in Figure 1. The log-rank test revealed a significant difference in the curves (P 〈 0.0001). Conclusions: Our data suggests that the use of T2* CMR, that enables individually tailored chelation regimes reducing the likelihood of developing decompensated cardiac failure, allowed the reduction of cardiac mortality in chronically transfused TM patients. Table 1. Table 1. Figure 1. Figure 1. Disclosures Pepe: Novartis: Speakers Bureau; Chiesi: Speakers Bureau; ApoPharma Inc: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.4567.4567
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
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  • 2
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    A Cost-Utility Analysis of Deferiprone Compared to Desferrioxamine and Deferasirox for the Treatment of Chronic Myocardial Iron Overload in Thalassemia Patients
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 2077-2077
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 2077-2077
    Abstract: Background: In thalassemia major (TM) three iron chelators are available to treat chronic iron overload due to blood transfusions: subcutaneous desferrioxamine (DFO), oral deferiprone (DFP) and oral deferasirox (DFX). Aims: This study evaluated the relative cost effectiveness of the three chelators in monotherapy. Methods: The cost-effectiveness model used is an Italian adaptation within the MIOT (Myocardial Iron Overload in Thalassemia) project of the previously built and published UK model (Bentley A et al. Pharmacoeconomics 2013;31:807-22). Based on literature and MIOT data, it was assumed that all the monotherapies had a comparable effect on and liver iron. Data about adverse events (AE) and cardiac morbidity were taken from the MIOT Network. Four different efficacy-based scenarios were explored in respect to cardiac morbidity and mortality using Markov-type models. Cost data were updated to reflect the Italian market: the tariffs applied in Veneto Region in the year 2014 were considered for the drugs, the administration of desferrioxamine and the monitoring. In Italy Veneto Region was proved to be one of the most upright region in the health costs management. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Results: Within the MIOT project, none of the AE considered in the UK model (neutropenia, agranulocytosis, Franconi syndrome, hepatitis) were detected for the 193 TM patients who had been received the same chelator for at least 18 months and were considered as reference group. The table shows costs and QALYs in the different modelled scenarios. For the first three scenarios a 5-year period was considered per patient while for the last scenario a 1-year time horizon was used and the discount rate of 3% was not applied. DFP was the dominant strategy in all scenarios, providing greater QALY at a lower cost. There was a higher QALY gain with DFX compared with DFO, but at a greater cost. Conclusions: The results of this analysis indicate that, from an Italian perspective, DFP is the most cost-effective treatment available for managing chronic iron overload in β-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings. Table 1. Drug cost Administration costs Monitoring costs AE costs Total costs QALYs Scenario 1: iron chelators impact cardiac morbidity and mortality. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €158,122 (€167,508) €0 (€0) €1,495 (€1,583) 0 €159,618 (€169,091) 3.876 (4.106) DFO €55,009 (€58,274) €12,034 (€12,748) €788 (€835) 0 €67,831 (€71,857) 3.285 (3.479) Scenario 2: iron chelators impact only cardiac mortality. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €158,122 (€167,508) €0 (€0) €1,495 (€1,583) 0 €159,618 (€169,091) 3.888 (4.119) DFO €55,009 (€58,274) €12,034 (€12,748) €788 (€835) 0 €67,831 (€71,857) 3.294 (3.490) Scenario 3: iron chelators impact only cardiac morbidity. Time horizon: 5 years. DFP €14,815 (€15,704) €0 (€0) €1,277 (€1,354) 0 €16,092 (€17,058) 3.962 (4.200) DFX €161,155 (€170,820) €0 (€0) €1,524 (€1,615) 0 €162,679 (€172,435) 3.951 (4.187) DFO €56,064 (€59,427) €12,264 (€13,000) €803 (€851) 0 €69,132 (€73,278) 3.348 (3.548) Scenario 4: iron chelators are all equivalent. Time horizon: 1 year. No discount. DFP €3,141 €0 €271 0 €3,412 0.840 DFX €34,164 €0 €334 0 €34,498 0.840 DFO €11,885 €2,600 €170 0 €14,656 0.712 Discounted valuea (Undiscounted value) Disclosures Pepe: ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau; Chiesi: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.2077.2077
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
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  • 3
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    Changes of Cardiac Iron and Function during Pregnancy in Trasfusion-Dependent Thalassemia Patients
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 3367-3367
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 3367-3367
    Abstract: Background. The aim of this study was to assess the changes in cardiac and hepatic iron overload and in morpho-functional cardiac parameters by Magnetic Resonance Imaging (MRI) in transfusion-dependent thalassemia patients who got pregnant and interrupted their chelation treatment. Methods. Among the956 women with hemoglobinopathies in reproductive age enrolled in the Myocardial Iron Overload in Thalassemia (MIOT) project, we selected 17 women with thalassemia (14 with thalassemia major and 3 with transfusion-dependent thalassemia intermedia) who had a pregnancy with successful delivery and who performed a MRI scan before and after the pregnancy. Myocardial and liver iron overload were measured by T2* multiecho technique. Atrial dimensions and biventricular function were quantified by cine images. Results. The pre-pregnancy MRI was performed 15.02±5.31 months before the delivery while the post-partum MRI was performed 5.73±4.45 months later. For 16 new-mothers the post-partum MRI was performed after the restart of the chelation therapy, specifically 3.95 ± 4.10 months later. One new-mother performed the post-partum MRI about 3 months before restarting the chelation therapy. The table shows the MRI parameters at the two MRIs. The pre-pregnancy and the post-partum global heart T2* values and number of pathological segments were comparable. Two patients with a normal global heart T2* value ( 〉 20 ms) before pregnancy showed a pathological post-partum value. After pregnancy there was a significant increase of MRI liver iron concentration (LIC) values. At the pre-partum MRI six (35.3%) patients had a MRI LIC 〈 3 mg/g/dw while at the post-partum MRI all patients had a pathological MRI LIC. Among the biventricular volumetric and functional parameters, there was a significant increase of right ventricular (RV) end-systolic volume index and a significant reduction of RV ejection fraction. Conclusion. In some transfusion-dependent patients, cessation of chelation therapy allows rapid iron overload. Pregnant women with thalassemia should be monitored carefully for iron loading and cardiac status before they embark upon a pregnancy and afterwards and consideration should be given to offering desferrioxamine chelation therapy immediately after delivery. In women showing severe iron overload before pregnancy desferrioxamine should be started after the middle of the second trimester. The negative impact on the RV parameters could reflect the effect of the high cardiac output state independent of the physiological changes during pregnancy. Table 1. Changes in MRI parameters following the pregnancy. Before pregnancy Post pregnancy Mean difference P-value Global Heart (ms) 33.27 ± 6.72 34.09 ± 9.46 0.82 ± 8.07 0.523 N seg. With T2* 〈 20 ms 1.71 ± 2.93 2.35 ± 4.72 0.65 ± 5.44 0.953 LIC (mg/g dw) 4.08 ± 3.55 16.89 ± 8.89 12.82 ± 8.19 〈 0.0001 LV EDVI (ml/m2) 76.53 ± 8.46 78.53 ± 10.42 2.00 ± 11.95 0.500 LV ESVI (ml/m2) 27.06 ± 3.96 29.24 ± 5.67 2.18 ± 5.37 0.114 LV SVI (ml/m2) 49.41 ± 7.19 47.41 ± 7.28 -2.00 ± 9.69 0.408 LV mass index (g/m2) 51.53 ± 8.43 54.76 ± 9.54 3.24 ± 6.66 0.062 LV EF (%) 64.00 ± 4.64 62.53 ± 4.68 -1.47 ± 5.86 0.317 RV EDVI (ml/m2) 73.24 ± 9.47 75.76 ± 10.94 2.53 ± 11.94 0.395 RV ESVI (ml/m2) 24.24 ± 6.06 27.82 ± 6.44 3.59 ± 6.43 0.035 RV SVI (ml/m2) 47.47 ± 8.35 47.41 ± 7.28 - 0.06 ± 10.69 0.982 RV EF (%) 66.82 ± 5.43 63.06 ± 5.51 3.77 ± 5.84 0.017 Disclosures Pepe: ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau; Chiesi: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.3367.3367
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 4
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    Deferiprone Has a Dose-Dependent Effect on Liver Iron Concentration
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 2159-2159
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 2159-2159
    Abstract: Purpose: The aim of this multi-centre study was to retrospectively assess in thalassemia major (TM) if deferiprone (DFP) had a dose-dependent effect on liver iron concentration (LIC) assessed by quantitative magnetic resonance imaging (MRI). Methods: Among the 958 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, we identified hose with an MRI follow up study at 18±3 months who had been received DFP monotherapy and had no changes in dose of DFP between the 2 MRI scans. Patients were divided into two groups according to the DFP dose: 79 patients with ≤ 75 mg/kg/d (group 1) and 39 with 〉 75 mg/kg/d (group 2). Hepatic iron overload was measured by the T2* multiecho technique and T2* values were converted into LIC values using the calibration curve introduced by Wood et al. Results: The two groups had comparable baseline MRI LIC values. The table shows the evolution of different iron overload risk classes between the baseline and the FU MRI. The percentage of patients that worsened their status was significantly higher in group 1 than in group 2 (26.6% vs 7.7%; P=0.016). Subgroup analysis in patients with hepatic iron overload at baseline (MRI LIC 〉 3mg/g/dw) was conducted: 48 patients from group 1 (DFP dose: mean 70.6±11.2 mg/kg/d, median 75 mg/kg/d) and 30 from group 2 (DFP dose: mean 85.2±6.6 mg/kg/d, median 84 mg/kg/d). The two subgroups had comparable baseline MRI LIC values (10.2±8.1 mg/g dw vs 11.1±8.7 mg/g dw (P=0.314). While the mean change in subgroup 2 ( -1.8±6.3mg/g/dw, P=0.131) was more favourable than in subgroup 1 (+0.1±7.7 mg/g/dw, P=0.903), the change in MRI LIC values did not reach statistical significance between the two subgroups (P=0.579) (Figure 1), which may be due to small cohort evaluated. Conclusions: In TM patients the worsening in MRI LIC can be prevented by increasing the dose of deferiprone above the widely used regimen of 75 mg/kg body weight. Our results are consistent with the iron balance studies performed by Grady RW et al. Table 1. Evolution of different iron overload risk classes between the baseline and the FU MRI. The underlined numbers represent the patients who remained in the same risk class. DFP dose ≤ 75 mg/kg/d (N=79) FU LIC 〈 3 mg/g dw 3-7 mg/g dw 7-15 mg/g dw ≥15 mg/g dw Baseline LIC 〈 3 mg/g dw (N=31) 21 7 3 0 3-7 mg/g dw (N=22) 10 4 6 2 7-15 mg/g dw (N=14) 0 6 5 3 ≥15 mg/g dw (N=12) 1 0 5 6 Total at the FU 32 17 19 11 DFP dose 〉 75 mg/kg/d (N=39) FU LIC 〈 3 mg/g dw 3-7 mg/g dw 7-15 mg/g dw ≥15 mg/g dw Baseline LIC 〈 3 mg/g dw (N=9) 6 2 1 0 3-7 mg/g dw (N=14) 3 11 0 0 7-15 mg/g dw (N=8) 0 4 4 0 ≥15 mg/g dw (N=8) 1 0 2 5 Total at the FU 10 17 7 5 Figure 1. Changes of MRI LIC values in patients with basal MRI LIC 〉 3 mg/g/dw. Figure 1. Changes of MRI LIC values in patients with basal MRI LIC 〉 3 mg/g/dw. Disclosures Pepe: Chiesi: Speakers Bureau; ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.2159.2159
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 5
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    Multi-Parametric Cardiac Magnetic Resonance for Prediction of Cardiac Complications in Thalassemia Intermedia: A Prospective Multicenter Study
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 4564-4564
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 4564-4564
    Abstract: Introduction: Cardiovascular Magnetic Resonance (CMR) has an established role in managing and predicting prognosis of patients with Thalassemia Major (TM). Thalassemia Intermedia (TI) is a milder variant of beta-thalassemia showing a different clinical and prognostic profile; pulmonary hypertension (PH) is a more common complication in TI patients. We prospectively determined the predictive value of CMR parameters, including measurement of right ventricular mass, for cardiac complications in TI. Methods: We considered 342 TI patients enrolled in the Myocardial Iron Overload in Thalassemia network; about half of them (178/302, 58.9%) were transfusion-dependent. Myocardial and liver iron overload were measured by T2* multiecho technique. Atrial dimensions, left and right ventricular mass and systolic function were quantified by cine images. Late gadolinium enhancement (LGE) images were acquired to detect myocardial fibrosis. Results: Twenty-three patients were excluded because a cardiac complication was present at the time of first CMR, so we prospectively followed 319 patients. All 319 patients were white, with a mean age at time of their first scan of 38.02±11.69 years and 165 (51.7%) of them were females. Mean follow-up time was 52.24±24.87 months (median 54.64 months). Cardiac events were recorded in 22 patients (6.9%): heart failure (HF) in 1 patient, arrhythmias in 12 patients, pulmonary hypertension (PH) in 7 patients and myocardial infarction (MI) in 2 patients. Due to the low number of events, only arrhythmias, PH and cardiac complications globally considered were taken as cardiac outcomes for univariate and multivariate analysis. In the multivariate analysis RV hypertrophy was the only independent predictive factor for arrhythmias (HR=33.83, 95% CI=6.07-188.74, P 〈 0.0001) and PH (HR=73.33, 95% CI=10.00-537.57, P 〈 0.0001). When cardiac complications were considered all together, RV hypertrophy (HR=24.12, 95% CI=5.09-114.12, P 〈 0.0001) and myocardial fibrosis by LGE (HR=6.59, 95% CI=1.33-32.67, P=0.021) were independent prognostic factors in the multivariate analysis. The Figures display the Kaplan-Meier curves showing the impact of the independent predictive factors on each outcome. Conclusions: For the first time we studied the prognostic value of right ventricular mass as part of multiparametric CMR imaging in a population of TI patients. RV hypertrophy identified patients at high risk for arrhythmias and PH. Both RV hypertrophy and fibrosis detected by LGE were independent predictive factor for cardiac complications. Measurement of RV mass should be part of the multi-parametric CMR study of patient with thalassemia intermedia. Figure 1. Figure 1. Disclosures Pepe: ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau; Chiesi: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.4564.4564
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
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  • 6
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    Prevalence of Extramedullary Hematopoiesis, Vertebral, Hepatic and Splenic Hemangioma and Renal Cyst Among Thalassaemic Patients: A Retrospective Study from the Myocardial Iron in Thalassemia (MIOT) Network
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 2154-2154
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 2154-2154
    Abstract: BACKGROUND: The prevalence and the clinical relevance of several extracardiac findings (EF) at Magnetic Resonance Imaging (MRI) have been recently estimated in cohorts of non thalassaemic patients. AIM: In this study we sought to determine the prevalence of EF potentially related to anaemia and ipoxia in age- and sex- matched populations with thalassaemia major (TM) and Thalassaemia Intermedia (TI). METHODS: We retrospectively considered 159 TM patients regularly transfused (73 F; 34±9.20 years) and 159 TI patients (73 F; 34±9.18 years) consecutively enrolled in the Myocardial Iron in Thalassemia (MIOT) Network to quantify cardiac and liver iron by T2* technique. Patients with TI were further subdivided in non transfusion dependent (NTD-TI) and transfusion dependent (TD-TI). All MRI images were used to assess the presence or absence of Extramedullary Hematopoiesis (EMH), kidney, splenic and liver cysts, and vertebral hemangiomas. RESULTS: Overall, EF were detected in 33% and 26% of patients with TI and TM, respectively. Table 1 shows the comparison of MRI data between TM and TI patients. Both groups of patients had elevated but comparable prevalence of kidney, splenic and liver cysts and vertebral haemangiomas. TI patients were found to have significant higher EMH as compared to TM. The prevalence of total EF increased with advancing age and renal cysts were found in the 28.6% of the cohort aged 45-55 years. Patients with renal cysts had comparable serum creatinin level with respect to those without. Within the population of TI-TD it was observed the highest prevalence of patients with renal cysts, splenic cyst and vertebral haemangioma (26%, 3% and 3%, respectively). Thalassemic patients had a was significant higher prevalence of renal cysts than the general population (19.2 vs 7.8%; P 〈 0.0001). CONCLUSIONS: EF are well-recognized features in patients undergoing MRI, being hepatic cysts or hemangiomas and renal cyst the most frequently observed. Our data on hemangiomas and renal cysts, particularly among patients with TI-TD, indicate a significant higher prevalence of EF compared to the general population. These data seem to suggest the role of the inappropriate activation of the Hypoxia-Inducible Factor (HIF) system linked to the chronic hypoxia as observed in the Chuvash polycythemia or in the the von Hippel-Lindau (VHL) syndrome. Furthermore, HIF in general population has been directly involved in the development and/or progression of clear cell renal cancer also described among thalassemic patients. Table 1. Table 1. Disclosures Pepe: Chiesi: Speakers Bureau; ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.2154.2154
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
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  • 7
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    Association Between Serum Ferritin and Liver Iron Concentration with Cardiac Iron in Pediatric Thalassemia Major Patients
    American Society of Hematology ; 2015
    In:  Blood Vol. 126, No. 23 ( 2015-12-03), p. 956-956
    Details
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 956-956
    Abstract: Introduction: Recently, the ability of LIC (liver iron concentration) and serum ferritin in predicting myocardial iron overload (MIO) has been challenged by magnetic Resonance Imaging (MRI) monitoring which demonstrated no or weak correlation between serum ferritin or LIC and MIO. Anyway, the role of this traditional markers could result particularly useful in pediatric population, where MRI assessment is difficult to carry out, because of early age, scarce collaboration or limited availability. So, we derived objective thresholds for these markers for predicting cardiac T2* 〈 20 ms in pediatric patients. Methods: From the 2171 patients with hemoglobinopathies enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, we retrospectively selected 107 paediatric patients with thalassemia major (TM) (61 boys, median age 14.4 years). MIO was assessed using a multislice multiecho T2* approach. Hepatic T2* values were assessed in a homogeneous tissue area and converted into LIC. Results: Twenty-three patients (21.5%) showed an abnormal global heart T2* value and none of them was under 7.9 years of age. Serum ferritin was negatively correlated with global heart T2* values (r=-0.425; P 〈 0.0001). Using ROC curve analysis, a serum ferritin of 2000 ng/ml was found to be the best threshold for discriminating the presence of cardiac iron with an area under the curve (AUC) of 0.733 (P=0.001) (Figure 1A) (Sensitivity= 0.73 and Specificity=0.65). Odds ratio (OR) for global heart T2* values 〈 20 ms was 4.9 (1.7-13.8 95%CI; P=0.003) for serum ferritin levels ≥2000 ng/ml. There was a significant negative correlation between global heart and MRI LIC values (P=-0.436; P 〈 0.0001). Using ROC curve analysis, a LIC≥14 mg/g/dw was found to be the best threshold for discriminating the presence of MIO in children with an AUC of 0.817 (P 〈 0.0001) (Figure 1B) (Sensitivity= 0.74 and Specificity=0.85 ). OR for abnormal global heart T2* values was 30.08 (3.58-252.68 95%CI; P=0.002) for patient with MRI LIC≥14 mg/g/dw versus patients with normal MRI LIC. Conclusion: A weak connection between serum ferritin levels or hepatic iron and cardiac iron was demonstrated in our pediatric population. Anyway, MRI LIC≥14 mg/g/dw and serum ferritin levels≥2000 ng/ml were found to be significant risk factors for a global heart T2* value 〈 20 ms in TM children. Figure 1. Figure 1. Disclosures Pepe: Novartis: Speakers Bureau; ApoPharma Inc: Speakers Bureau; Chiesi: Speakers Bureau.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V126.23.956.956
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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