A T2* MRI Prospective Survey on Pancreatic Iron in Thalassemia Major Patients Treated with Deferasirox, Deferiprone and Desferrioxamine

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4044-4044
Author(s):  
Antonella Meloni ◽  
Cristina Paci ◽  
Giuseppe Serra ◽  
Stefania Vacquer ◽  
Roberto Giugno ◽  
...  

Abstract Background. Impairment of the endocrine and exocrine function of the pancreas is a common complication in thalassemia major (TM). Multiecho T2* Magnetic Resonance Imaging (MRI) allows the reproducible and noninvasive assessement of pancreatic iron overload. However, there are no prospective studies describing the changes of pancreatic T2* values. So, our aim was to describe the changes in pancreatic T2* values over a follow-up (FU) of 18 months and to evaluate prospectively the effectiveness of the three iron chelators in monotherapy. Methods. We selected 22 TM patients consecutively enrolled in the Myocardial Iron Overload in Thalassemia (MIOT) Network who had received only one chelator in monotherapy between the two MRI scans. Pancreatic iron burden was measured using a T2* gradient-echo multiecho sequence. The images were analyzed using a previously validated, custom-written software (HIPPO-MIOT®). Results. Three groups of patients were identified: 9 patients (5 females, mean age 32.8±8.6 years) treated with desferioxamine (DFO – mean dosage 44.8±3.8 mg/kg/die), 6 patients (2 females, mean age 36.3±6.5 years) treated with deferiprone (DFP– dosage 75mg/kg/die) and 7 patients (6 females, mean age 30.4±9.1 years) treated with deferasirox (DFX – mean dosage 28.2±4.6 mg/kg/die). The percentage of patients with a excellent/good compliance was comparable among the groups. All patients under DFO therapy showed at the baseline MRI pancreatic iron overload (T2*<26 ms) and at the FU only one had a normal pancreas T2* value (Figure 1, left). Globally there was a significant increment in the pancreatic T2* values (mean difference: 3.66±3.94; P=0.021). In the DFP group at baseline 5 patients showed pancreatic iron and none recovered at the follow up (Figure 1, center). The patient with a normal baseline pancreatic T2* value, maintained it at the FU. For the subgroup with pancreatic iron at the baseline, there was a significant increment in the pancreatic T2* values (mean difference: 3.99±2.05; P=0.043). In the DFX group 5 patients showed at the baseline pancreatic iron and although the pancreatic T2* increased for all of them, the normal value was not reached at the follow up (Figure 1, right). Both the patients who showed no pancreatic iron overload at the baseline maintained at the FU the same status. For the subgroup with pancreatic iron at the baseline, there was a significant increment in the pancreatic T2* values (mean difference: 2.48±3.06; P=0.043). Conclusion: Prospectively in TM patients at the dosages used in the clinical practice all three chelators in monotherapy allowed a significant reduction in pancreatic iron. Further prospective studies involving more patients are needed to establish which is the most effective drug. Figure 1 Figure 1. Disclosures Pepe: Novartis: Speakers Bureau; ApoPharma Inc.: Speakers Bureau; Chiesi: Speakers Bureau.

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4901-4901
Author(s):  
Antonella Meloni ◽  
Lorella Pitrolo ◽  
Blandina Pagano ◽  
Giancarlo Izzi ◽  
Augusto Scaccetti ◽  
...  

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.


2021 ◽  
Vol 7 (1) ◽  
pp. 205521732199239
Author(s):  
Cecilie Jacobsen ◽  
Robert Zivadinov ◽  
Kjell-Morten Myhr ◽  
Turi O Dalaker ◽  
Ingvild Dalen ◽  
...  

Objectives To identify Magnetic Resonance Imaging (MRI), clinical and demographic biomarkers predictive of worsening information processing speed (IPS) as measured by Symbol Digit Modalities Test (SDMT). Methods Demographic, clinical data and 1.5 T MRI scans were collected in 76 patients at time of inclusion, and after 5 and 10 years. Global and tissue-specific volumes were calculated at each time point. For the primary outcome of analysis, SDMT was used. Results Worsening SDMT at 5-year follow-up was predicted by baseline age, Expanded Disability Status Scale (EDSS), SDMT, whole brain volume (WBV) and T2 lesion volume (LV), explaining 30.2% of the variance of SDMT. At 10-year follow-up, age, EDSS, grey matter volume (GMV) and T1 LV explained 39.4% of the variance of SDMT change. Conclusion This longitudinal study shows that baseline MRI-markers, demographic and clinical data can help predict worsening IPS. Identification of patients at risk of IPS decline is of importance as follow-up, treatment and rehabilitation can be optimized.


Author(s):  
Martina Pecoraro ◽  
Stefano Cipollari ◽  
Livia Marchitelli ◽  
Emanuele Messina ◽  
Maurizio Del Monte ◽  
...  

Abstract Purpose The aim of the study was to prospectively evaluate the agreement between chest magnetic resonance imaging (MRI) and computed tomography (CT) and to assess the diagnostic performance of chest MRI relative to that of CT during the follow-up of patients recovered from coronavirus disease 2019. Materials and methods Fifty-two patients underwent both follow-up chest CT and MRI scans, evaluated for ground-glass opacities (GGOs), consolidation, interlobular septal thickening, fibrosis, pleural indentation, vessel enlargement, bronchiolar ectasia, and changes compared to prior CT scans. DWI/ADC was evaluated for signal abnormalities suspicious for inflammation. Agreement between CT and MRI was assessed with Cohen’s k and weighted k. Measures of diagnostic accuracy of MRI were calculated. Results The agreement between CT and MRI was almost perfect for consolidation (k = 1.00) and change from prior CT (k = 0.857); substantial for predominant pattern (k = 0.764) and interlobular septal thickening (k = 0.734); and poor for GGOs (k = 0.339), fibrosis (k = 0.224), pleural indentation (k = 0.231), and vessel enlargement (k = 0.339). Meanwhile, the sensitivity of MRI was high for GGOs (1.00), interlobular septal thickening (1.00), and consolidation (1.00) but poor for fibrotic changes (0.18), pleural indentation (0.23), and vessel enlargement (0.50) and the specificity was overall high. DWI was positive in 46.0% of cases. Conclusions The agreement between MRI and CT was overall good. MRI was very sensitive for GGOs, consolidation and interlobular septal thickening and overall specific for most findings. DWI could be a reputable imaging biomarker of inflammatory activity.


2018 ◽  
Vol 37 (01) ◽  
pp. 54-57
Author(s):  
Renato Pignatari ◽  
Manoel Teixeira ◽  
Eberval Figueiredo

AbstractThe pilomyxoid astrocytoma (PMA) is a rare glioma recently described as a separate entity, which is generally located on the hypothalamic area. The PMA was previously described as pilocytic astrocytoma (PA) due to similarities shared between them. Recent studies provided a deeper understanding of PMA, setting it as a separate entity, though PMA is still considered by many authors a variant of PA. The PMA is considered to be more aggressive than PA; however, further studies are necessary for a better comprehension of its behavior and, hence, for neurosurgeons and neurologists to get to a consensus about its management.This study presents a 16-year-old female patient who looked for medical assistance complaining of headaches of over 6 months and vomiting for 2 weeks prior to the visit to the doctor. She presented no other symptoms. The physical examination displayed only bilateral papilledema. The magnetic resonance imaging (MRI) scans showed an intraventricular and thalamic lesion composed of solid and cystic material associated with peritumoral edema. The patient underwent ventriculoperitoneal shunt and subtotal resection of the lesion. The histological and immunohistochemical studies showed typical features of PMA. The patient started adjuvant therapy with chemotherapy and radiosurgery. She has been asymptomatic for 9 months and has shown no signs of progression of the disease on the follow-up scans.


2019 ◽  
Vol 34 (10) ◽  
pp. 586-592
Author(s):  
Emily W.Y. Tam ◽  
Vann Chau ◽  
Raphaël Lavoie ◽  
M. Mallar Chakravarty ◽  
Ting Guo ◽  
...  

To help clinicians understand what to expect from small cerebellar volumes after prematurity, this study aims to characterize the specific impacts of small cerebellar volumes on the infant neurologic examination. A prospective cohort of preterm newborns (<32 weeks’ gestational age) had brain magnetic resonance imaging (MRI) studies at term-equivalent age. Cerebellar volumes were compared with neurologic examination findings in follow-up, adjusting for severity of intraventricular hemorrhage, white matter injury, and cerebellar hemorrhage. Deformation-based analyses delineated regional morphometric differences in the cerebellum associated with these findings. Of 119 infants with MRI scans, 109 (92%) had follow-up at 19.0±1.7 months corrected age. Smaller cerebellar volume at term was associated with increased odds of truncal hypotonia, postural instability on standing, and patellar hyperreflexia ( P < .03). Small cerebellar volume defined as <19 cm3 by 40 weeks was associated with 7.5-fold increased odds of truncal hypotonia ( P < .001), 8.9-fold odds postural instability ( P < .001), and 9.7-fold odds of patellar hyperreflexia ( P < .001). Voxel-based deformation-based morphometry showed postural instability associated with paravermian regions. Small cerebellar volume is associated with specific abnormalities on neurologic examination by 18 months of age, including truncal tone, reflexes, and postural stability.


Author(s):  
Nashwa El-Khazragy ◽  
Safa Matbouly ◽  
Demiana H. Hanna ◽  
Nievin Ahmed Mahran ◽  
Sally Abdallah Mostafa ◽  
...  

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2771-2771
Author(s):  
Wing Y. Au ◽  
Wynnie Lam ◽  
Winnie Chu ◽  
Hui-leung Yuen ◽  
S.C. Ling ◽  
...  

Abstract Background: The use of magnetic resonance imaging (MRI) for organ specific iron assessment has allowed better tailoring of chelation therapy. Since endocrine failure is common in thalassemia major (TM) patients, we explored the utility of rapid T2* MRI assessment of hemosiderosis in the pancreas and pituitary. The results were correlated with standard T2* heart and liver MRI assessments and clinical data. Material and methods: A total of 180 TM patients (M:F = 91:89, median age, range 12–48) were scanned on a 1.5 T scanner. (Sonata, Siemens Medical, Erlanger, Germany). T2* myocardium was assessed by a cardiac gated single breath hold 8-echo sequence (CMRtools; London, UK). The T2* liver, pancreas and pituitary were performed by a breath hold 20-echo sequence. Subcutaneous deferoxamine was used for chelation, except for addition of deferiprone in 24 cases for 1 year. Results: There was a high incidence of hemosiderosis of heart (severe T2*<12ms: 34%, mild-moderate <20ms, 15%) and liver (severe T2*<1.4ms, 14% mild-moderate <6.3ms, 63%). Iron overload above normal control was commonly found in the pancreas (T2*<23ms, 84%) and pituitary (T2*<5.9ms, 24%). Pancreatic T2* correlated with pituitary T2* (p=0.007, r=0.2), cardiac T2*(p<0.001, r=0.33), liver T2* (p<0.001, r=0.35), ferritin (p=0.004, r=−0.19) and age (p=0.033, r=0.16). Similarly pituitary T2* related to cardiac T2* (p<0.001, r=0.36) and liver T2* (p=0.026, r=0.17). On multivariate analysis, however, pancreatic T2* related to both heart T2* (p<0.001) and liver T2* (p=0.001), while pituitary T2* only related to heart T2* (p<0.001). Documented complications amongst the cases included heart failure (ejection fraction EF<55%, n=28, 16%), hypogonadism (n=84,47%), diabetes mellitus (n=44, 25%), hypoparathyroidism (n=16, 9%) and hypothyroidism (n=36, 20%), with hepatitis B and C carrier state in 2% and 25% respectively. On univariate and multivariate analysis, all 4 endocrine failures correlated with only cardiac T2* results (all p<0.001) and age (all p<0.001), but not with pituitary or pancreatic T2* results. The EF correlated with T2* of pituitary, pancreas and heart, but only MRI heart T2* correlation remained significant on multivariate analysis. Conclusions: MRI pituitary and pancreatic evaluation is viable in a cohort of poorly chelated Chinese thalassemia major patients on subcutaneous deferoxamine treatment. However, an abnormal cardiac T2* result is a good surrogate for endocrine iron overload and appeared more relevant in predicting endocrine complications.


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5165-5165
Author(s):  
Alessia Pepe ◽  
Giuseppe Rossi ◽  
Antonella Meloni ◽  
Dell'Amico Maria Chiara ◽  
D'Ascola Domenico Giuseppe ◽  
...  

Abstract Abstract 5165 Introduction: Most deaths in thalassemia major (TM) result from cardiac complications due to iron overload. No data are available in literature about possible different changes in cardiac and liver iron in TM patients treated with sequential deferiprone–deferoxamine (DFP-DFO) versus deferasirox (DFX). Magnetic Resonance (MR) is the unique non invasive suitable technique to evaluated quantitatively this issue. The aim of this multi-centre study was to assess prospectively in the clinical practice the efficacy of the DFP-DFO vs DFX in a cohort of TM patients by quantitative MR. Methods: Among the first 739 TM patients enrolled in the MIOT (Myocardial Iron Overload in Thalassemia) network, 253 patients performed a MR follow up study at 18 ± 3 months according to the protocol. We evaluated prospectively the 25 patients treated with DFP-DFO versus the 44 patients treated with DFX between the 2 MR scans. Myocardial and liver iron concentrations were measured by T2* multislice multiecho technique. Results: The doses of the sequential treatment were DFP 70±14 mg/kg/d for 4 d/w and DFO 42±8 mg/kg/d for 3 d/w, the dose of DFX was 26±6 mg/kg/d. Excellent/good levels of compliance were similar in the 2 groups (DFP-DFO 96% vs DFX 100%; P = 0.36). At baseline the 2 groups were homogeneous for cardiac and liver iron. Among the patients with no significant myocardial iron overload at baseline (global heart T2* 3 20 ms), there were no significant differences between groups to maintain the patients without myocardial iron overload (DFP-DFO 95% vs DFX 96%; P = 1.0). Among the patients with myocardial iron overload at baseline (global heart T2* < 20 ms), only in the DFX group there was a significant improvement in the global heart T2* value (11 ± 5 ms at baseline versus 16 ± 8 at 18 ± 3 months, P = 0.0001) and in the number of segment with a normal T2* value (P = 0.003). The improvement in the global heart T2* was not significantly difference in the DFP-DFO versus the DFX group (mean difference global heart T2* 2.2 ± 4.1 ms versus 4.6 ± 4.8 P = 0.2). The changes in the mean serum ferritin level were not significantly different between groups. In patients with liver iron overload at baseline (liver T2* < 5.1 ms), the change in the liver T2* was not significant between groups (mean difference liver T2* 0.9 ± 2.1 ms vs 2.4 ± 5.2; P = 0.3). Conclusions: Prospectively in the clinical setting over 15 months we did not find significant differences on cardiac and liver iron by quantitative MRI in TM patients treated with sequential DFP–DFO versus the TM patients treated with DFX. Disclosures: No relevant conflicts of interest to declare.


2014 ◽  
Vol 20 (14) ◽  
pp. 1912-1915 ◽  
Author(s):  
L D’Anna ◽  
S Lorenzut ◽  
A Perelli ◽  
B Zanchettin ◽  
M Valente ◽  
...  

Radiologically-isolated syndrome (RIS) is a recently-defined entity, described as the incidental discovery of lesions suggestive of multiple sclerosis (MS) on brain magnetic resonance imaging (MRI) scans demonstrating dissemination in space (DIS) without symptom expression and with a normal neurological examination. Recent studies demonstrate that RIS patients present similar features of cognitive impairment as MS patients. We describe a case of a RIS patient in whom investigating cognitive functions was a useful tool for diagnostic and therapeutic decisions.


2016 ◽  
Vol 22 (9) ◽  
pp. 1174-1183 ◽  
Author(s):  
Mike P Wattjes ◽  
Martijn T Wijburg ◽  
Anke Vennegoor ◽  
Birgit I Witte ◽  
Stefan D Roosendaal ◽  
...  

Background: In natalizumab-treated multiple sclerosis (MS) patients, magnetic resonance imaging (MRI) is considered as a sensitive tool in detecting both MS disease activity and progressive multifocal leukoencephalopathy (PML). Objective: To investigate the performance of neuroradiologists using brain MRI in detecting new MS lesions and asymptomatic PML lesions and in differentiating between MS and PML lesions in natalizumab-treated MS patients. The secondary aim was to investigate interrater variability. Methods: In this retrospective diagnostic study, four blinded neuroradiologists assessed reference and follow-up brain MRI scans of 48 natalizumab-treated MS patients with new asymptomatic PML lesions ( n = 21) or new MS lesions ( n = 20) or no new lesions ( n = 7). Sensitivity and specificity for detection of new lesions in general (MS and PML lesions), MS and PML lesion differentiation, and PML detection were determined. Interrater agreement was calculated. Results: Overall sensitivity and specificity for the detection of new lesions, regardless of the nature of the lesions, were 77.4% and 89.3%, respectively; for PML-MS lesion differentiation, 74.2% and 84.7%, respectively; and for asymptomatic PML lesion detection, 59.5% and 91.7%, respectively. Interrater agreement for the tested categories was fair to moderate. Conclusion: The diagnostic performance of trained neuroradiologists using brain MRI in pharmacovigilance of natalizumab-treated MS patients is moderately good. Interrater agreement among trained readers is fair to moderate.


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