scholarly journals Evaluation of iron overload by cardiac and liver T2* in β-thalassemia: Correlation with serum ferritin, heart function and liver enzymes

2021 ◽  
Vol 13 (1) ◽  
pp. 54-60
Author(s):  
Hengameh Khadivi Heris ◽  
Babak Nejati ◽  
Khatereh Rezazadeh ◽  
Hossein Sate ◽  
Roya Dolatkhah ◽  
...  
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Liver Enzymes ◽  
Heart Function ◽  
Ferritin Level ◽  
Diagnostic Methods ◽  
Iron Load ◽  
The Relationship ◽  
T2 Mri

Introduction: In this study, we aimed to assess the relationship of cardiac and hepatic T2* magnetic resonance imaging (MRI) values as a gold standard for detecting iron overload with serum ferritin level, heart function, and liver enzymes as alternative diagnostic methods. Methods: A total 58 patients with beta-thalassemia major who were all transfusion dependent were evaluated for the study. T2* MRI of heart and liver, echocardiography, serum ferritin level, and liver enzymes measurement were performed. The relationship between T2* MRI findings and other assessments were examined. Cardiac and hepatic T2* findings were categorized as normal, mild, moderate, and severe iron overload. Results: 22% and 11% of the patients were suffering from severe iron overload in heart and liver, respectively. The echocardiographic findings were not significantly different among different iron load categories in heart or liver. ALT level was significantly higher in patient with severe iron overload than those with normal iron load in heart (P=0.005). Also, AST level was significantly lower in normal iron load group than mild, moderate, and severe iron load groups in liver (P<0.05). The serum ferritin level was significantly inversely correlated with cardiac T2* values (r = -0.34, P=0.035) and hepatic T2* values (r = -0.52, P=0.001). Conclusion: Cardiac and hepatic T2* MRI indicated significant correlation with serum ferritin level.

scholarly journals Correlation between serum ferritin level, cardiac and hepatic T2-star MRI in patients with β-thalassemia major in Al-Diwaniya thalassemia center

2018 ◽  
Vol 9 (SPL1) ◽  
Author(s):  
Alaa Mutter Jabur Al-Shibany ◽  
AalanHadi AL-Zamili
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Iron Level ◽  
The Mean ◽  
T2 Mri

Patients with transfusion dependent thalassemia major is often associated with iron overload. Proper use of iron chelators to treat iron overload requires an accurate measurement of iron levels. Magnetic resonance T2-star (T2* MRI) is the preferred method to measure iron level in the liver andthe heart. The goal of our study was to see if there is an association exists between serum ferritin level and T2* MRI results in patients with beta thalassemia major.This study was done in Al-Diwaniya Thalassemia center,Maternity and children teaching hospital,Iraq. During the period from 1st of January to 31st of October. Fifty eight patients with a diagnosis of beta thalassemia major were enrolled in the study. They were older than five years old,transfusion dependent and on chelation therapy. Hepatic and Myocardial T2*MRI and the mean serum ferritin levels were measured during the study period for all patients.There is a significant correlation was observed between serum ferritin level and cardiac T2*MRI (p=0.018 ). also a significant correlation was observed between serum ferritin and hepatic T2*MRI (p=0.02). Neither cardiac T2* MRI nor hepatic T2* MRI show any correlation with the mean age.our study also showa positive correlation between the patients withcardiac T2* MRI and the development of diabetes mellitus in contrast to hepatic T2* MRI in which there is no any correlation. Hypothyroidism was observedno correlation with either cardiac or hepatic T2* MRI.Our results showed a positiveassociation between hepatic, cardiac T2*MRI and serum ferritin levels.

scholarly journals Transient Elastography of Liver in Hb E Beta Thalassemia: A Novel Tool to Determine Hepatic Iron Overload?

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3368-3368
Author(s):  
Debmalya Bhattacharyya ◽  
Maitreyee Bhattacharyya ◽  
Saswata Chatterjee ◽  
Abhijit Chowdhury ◽  
Pramit Ghosh
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Liver Stiffness ◽  
Beta Thalassemia ◽  
Hepatic Iron ◽  
Hepatic Iron Overload ◽  
Number Of Patients ◽  
T2 Mri

Abstract Introduction: Transient Elastography (TE) of liver is a well established tool to measure liver stiffness, mainly used for assessment of hepatic fibrosis due to chronic hepatitis. Liver biopsy is the gold standard test for measurement of liver iron concentration (LIC) whereas T2* MRI is the best available non-invasive method for the same in thalassemia. We intended to use hepatic TE as an alternative cheaper tool to assess hepatic iron overload so that it can be applied to larger number of patients. Objective: To assess degree of liver stiffness by TE in patients with HbE beta thalassemia and correlate the findings with LIC calculation by T2* MRI of liver. Materials and Method: 53 patients with HbE beta thalassemia from the thalassemia clinic of Institute of Haematology and Transfusion Medicine, Medical College, Kolkata were enrolled for the study. Patients with known liver disease were excluded. Baseline data like HbE%, mutations, transfusion requirement, growth status, serum ferritin level etc were collected. All of them underwent TE of liver in the School of Digestive and Liver Diseases, IPGMER using the FibroScan Touch 502 machine (Di Marco et al, British Journal of Haematology, Volume 148,3, 476-479, February 2010). 20 randomly selected patients were also assessed by T2*MRI of liver for hepatic iron assessment at the same time. LIC calculation was done from T2* value (J S Hankins et al, Blood, 14 May 2009, Volume 113:20). Data were analyzed by SPSS software-19, IBM. Results: The patients with HbE beta thalassemia had a mean HbE level of 53.66 (±18.45) %. Common beta mutations [mostly IVS-1-5(G-C)] usually found in this part of India, were detected. Mean and median age of the study population was 24.11±13.11 years and 20 years, respectively. Median age of 1st transfusion was 11 years. 35.84% patients were non-transfusion dependent. 39/53 patients had facial deformity and growth retardation. Mean baseline hemoglobin was 7.10±0.76 gm/ dl. Mean serum ferritin level was 3183.66±338.45 ng/ml. TE showed 30.18 % patients had severe liver stiffness (Liver stiffness measurement, LSM >15 kPa) whereas 43.34% had minimum stiffness (LSM≤7 kPa). No significant statistical correlation was found between serum ferritin and LSM. 12/20 patients showed very high calculated LIC (>15 mg/g) and lower T2* value (<1.8 ms) whereas only 10% of them showed mildly elevated calculated LIC. Rest had intermediate LIC. Discussion: There is lack of data regarding hepatic iron overload in HbE beta thalassemia and so also from this part of India. There was a trend that higher the age, higher was the LSM irrespective of the serum ferritin level though not found statistically significant (Figure 1). Serum ferritin level was also not significantly correlated with the calculated LIC in those 20 patients assessed with T2* MRI. 2 patients with mildly elevated LIC had a high ferritin level. Preliminary report indicates that with increase in LSM there was increase in calculated LIC also. Statistical analysis revealed patients with LSM≥7.2 kPa had moderate or severe hepatic iron overload and thus undermine the need for routine T2*MRI. The cut off value signifies that patients with LSM<7.2 kPa might or might not have significantly high liver iron overload, so obviously to be assessed by T2*MRI (Table 1). Therefore use of TE may be an alternative preliminary diagnostic method to gauge hepatic iron overload in HbE beta thalassemia patients. It would be of more value in countries like India where T2* MRI facility is not yet feasible in many centers catering to huge number of HbE-beta thalassemia patients. However, further exploration with larger number of patients is necessary to establish association of LIC and LSM in a more robust way. Conclusion: In resource-poor countries like India, TE may be a relatively cheap tool to be used as a marker of hepatic iron overload in future. Table 1. Finding Cut off: ROC (TE-value and LIC categories), n=20 Positive if Greater Than or Equal Toa Sensitivity 1 - Specificity 2.3 1.00 1.00 3.4 1.00 .50 4.4 .94 .50 5.7 .88 .50 6.2 .83 .50 6.5 .77 .50 7.2 .77 .00 8.2 .72 .00 8.85 .66 .00 9.45 .61 .00 10.2 .55 .00 11.85 .50 .00 13.85 .44 .00 15.75 .38 .00 18.3 .33 .00 22.9 .27 .00 27.9 .22 .00 35.9 .16 .00 44.7 .11 .00 48.0 .05 .00 49.8 .00 .00 Table 2. The smallest cutoff value is the minimum observed test value minus 1, and the largest cutoff value is the maximum observed test value plus 1. LSM more than 7.2 had a sensitivity of 77.2 % and specificity of 100%. Figure 2. Figure 2. Disclosures No relevant conflicts of interest to declare.

scholarly journals Noninvasive assessment of iron overload by magnetic resonance imaging

2020 ◽  
Vol 19 (3) ◽  
pp. 158-163
Author(s):  
E. E. Nazarova ◽  
D. A. Kupriyanov ◽  
G. A. Novichkova ◽  
G. V. Tereshchenko
Keyword(s):  
Magnetic Resonance Imaging ◽  
Magnetic Resonance ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Iron Concentration ◽  
The Body ◽  
Iron Accumulation ◽  
Resonance Imaging

The assessment of iron accumulation in the body is important for the diagnosis of iron overload syndrome or planning and monitoring of the chelation therapy. Excessive iron accumulation in the organs leads to their toxic damage and dysfunction. Until recently iron estimation was performed either directly by liver iron concentration and/or indirectly by measuring of serum ferritin level. However, noninvasive iron assessment by Magnetic resonance imaging (MRI) is more accurate method unlike liver biopsy or serum ferritin level test. In this article, we demonstrate the outlines of non-invasive diagnostics of iron accumulation by MRI and its specifications.

scholarly journals Red meat and egg intake and serum ferritin concentrations in Colombian children: results of a population survey, ENSIN-2015

2020 ◽  
Vol 9 ◽  
Author(s):  
Oscar F. Herran ◽  
Jhael N. Bermúdez ◽  
María Del Pilar Zea
Keyword(s):  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Red Meat ◽  
Cross Sectional ◽  
Egg Consumption ◽  
Dietary Consumption ◽  
The Mean ◽  
The Relationship ◽  
Meat And Eggs

Abstract The present study aimed to (a) establish the frequency of consumption of red meat and eggs; (b) determine serum ferritin levels (μg/l); and (c) establish the relationship between serum ferritin and the consumption of red meat and eggs. In Colombia during 2014–2018, an analytical study was conducted in 13 243 Colombian children between the ages of 5 and 17 years, based on cross-sectional data compiled by ENSIN-2015 (Encuesta Nacional de la Situación Nutricional en Colombia-2015) on serum ferritin levels and dietary consumption based on a questionnaire of the frequency of consumption. Using simple and multiple linear regression, with the serum ferritin level as the dependent variable and the frequency of consumption as the main explanatory variable, the crude and adjusted partial regression coefficients (β) between serum ferritin levels and consumption were calculated. The frequency of habitual consumption of red meat was 0⋅49 (95 % CI 0⋅47, 0⋅51) times/d. The frequency of habitual egg consumption was 0⋅76 (95 % CI 0⋅74, 0⋅78) times per d. The mean serum ferritin level in men was 41⋅9 (95 % CI 40⋅6, 43⋅1) μg/l and in women, 35⋅7 (95 % CI 34⋅3, 37⋅7) μg/l (P < 0⋅0001). The adjusted β between the consumption of red meat and eggs and serum ferritin levels were β = 3⋅0 (95 % CI 1⋅2, 4⋅7) and β = 2⋅5 (95 % CI 1⋅0, 3⋅9) for red meat and eggs, respectively. In conclusion, red meat and eggs are determinants of serum ferritin levels in Colombia and, therefore, could be considered public policy options to reduce anaemia and Fe deficiency.

scholarly journals Tissue doppler imaging in thalassemia major patients: correlation between systolic and diastolic function with serum ferritin level

2012 ◽  
Vol 52 (4) ◽  
pp. 187 ◽  
Author(s):  
Syarif Rohimi ◽  
Najib Advani ◽  
Sudigdo Sastroasmoro ◽  
Bambang Mardiyono ◽  
Sukman Tulus Putra ◽  
...  
Keyword(s):  
Iron Overload ◽  
Serum Ferritin ◽  
Tissue Doppler Imaging ◽  
Negative Correlation ◽  
Serum Ferritin Level ◽  
Systolic Function ◽  
Ferritin Level ◽  
Tissue Doppler ◽  
Doppler Imaging ◽  
Number Of Patients

Background Thalassemia is a major public health problem inIndonesia. Cardiac diseases remain as the main cause of death inthese patients due to iron overload. Although the T2* magneticresonance imaging has been considered as the gold standard forassessing cardiac iron overload but it has limited availability.The tissue doppler imaging (TDI) echocardiography, a fairly newand easy method that is suggested, can detect early abnormalmyocardial iron overload.Objective To assess myocardial systolic and diastolic functionof thalassemic patients using TDI and examine their correlationwith serum ferritin level.Methods A cross􀁌sectional study was conducted from January toMarch 2011 at the Harapan Kita Women and Children Hospital.We performed clinical examination, serum ferritin level, as wellas conventional and tissue doppler echocardiography on allsubjects.Results We included 34 regularly􀁌tranfused patients, of which17 were boys. The mean age of the subjects was 11.6 (SD 4.7years, range 2.6 􀁌 20 years). Mean pulse rate and blood pressurewere within normal range. Hemoglobin level at inclusion rangedfrom 5.8 to 6 g/dL. Almost all patients did not receive regularchelation therapy. Median serum ferritin level was 6275 ng/mL(range 2151 - 17,646 ng/mL). Conventional echocardiographyshowed normal systolic function, but some diastolic dysfunctionswere found including E wave abnormalites in 4 patients, A waveabnormalites in 3, and E/A ratio abnormalites found in 3. TheTDI showed decreased systolic function (Sa wave abnormality) in9 patients and diastolic dysfunctions (Ea wave abnormality in 11patients and Aa wave abnormaly in 2). No abnormality was foundin Ea/Aa and ElEa ratios. There was a weak negative correlationbetween ferritin level and Sa wave and Ea wave respectively anda moderately negative correlation between ferritin level and Ea/Aa ratio. There was no correlation between serum ferritin andAa wave or ElEa ratio.Conclusion TDI identifies a greater number of patients Mthsystolic and diastolic myocardial dysfunction than was revealedby conventional echocardiography. There was a weak negativecorrelation between serum ferritin to Sa wave and Ea wave, and amoderately negative correlation between ferritin and Ea/Aa ratio.There was no correlation between serum ferritin and Aa wave orElEa ratio. [paediatr Indones. 2012;52:187,93].

Hematologic Improvement with Iron Chelation using therapy Deferasirox in Patients with Aplastic Anemia: A Subgroup Analysis of KAMS0112 Prospective Study,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3424-3424
Author(s):  
Yoo-Hong Min ◽  
Sung-Soo Yoon ◽  
Hyeoung Joon Kim ◽  
Kyoo-Hyung Lee ◽  
Jae Hoon Lee ◽  
...  
Keyword(s):  
Platelet Count ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Research Funding ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Hemoglobin Level ◽  
Hematopoietic Stem ◽  
Wbc Count

Abstract Abstract 3424 Patients with aplastic anemia (AA) are suffered from various complications related to bone marrow failure and peripheral cytopenia. Although immunosuppressive therapy or hematopoietic stem cell transplantation has been performed for curative purpose, the majority of patients have been treated only by supportive cares including repeated transfusion. However, because continued transfusion eventually induces iron overload in many tissues and organs, transfusional iron overload and its consequences are another life-threatening problems for AA patients. Previous reports about iron chelation therapy (ICT) have mainly shown its efficiency for decreasing tissue iron and safety. However, improvement in hematopoiesis after iron chelation therapy has been limitedly reported as case reports or trials involving small number of patients without objective tools for measuring tissue iron content. In the KAMS0112 study (a multi-center, open label, prospective study evaluating the efficacy of ICT with deferasirox in transfusional iron overload with myelodysplastic syndrome or AA using quantitative R2-MRI, Ferriscan), a total of 54 patients with AA showing serum ferritin level over 1,000 ng/ml were enrolled from 19 institutes, and further analyzed for the changes in hemogram during ICT as well as efficacy and safely of deferasirox. During the study, the specific treatments for AA, such as immunosuppressive therapy or hematopoietic stem cells transplantation, were not undertaken. During 1 year prior to study, patients received 23.7±16.9 units of red blood cell (RBC) product, and the baseline serum ferritin level and liver iron content (LIC) were 4,164±447 ng/ml and 20.1±12.0 mg Fe/g DW, respectively. Deferasirox was given orally at a dose of 20 mg/kg/day for at least 6 months to all patients. If the serum ferritin level falls below 500 ng/ml, treatment was withheld. In spite of continued transfusional support during the study, serum ferritin level and LIC were significantly decreased after 1 year of ICT with deferasirox (Ds-ferritin=−3,076.7±489.9 ng/ml, p=0.0003; DLIC=−7.73 mg/Fe/g DW, p=0.001). To evaluate the improvement of each parameter in hemogram by ICT, patients with baseline hemoglobin level less than 8.0 g/dl (n=28), with baseline WBC count less than 4/ml (n=43), and with baseline platelet count less than 20/ml (n=31) were selected separately. At the end of study, hemoglobin level and platelet count (8.2±3.0 g/dl and 22.2±31.4/ml, respectively) was significantly increased from the baseline value (6.1±1.1 g/dl, p=0.001; 12.5±12.4/ml, p=0.05, respectively). WBC count was also slightly increased (from 2.1±0.9/ml to 2.3±0.9/ml, p=0.457). Considering the relatively uniform criteria of transfusion, the finding that hemoglobin level and platelet count could increase above 8 g/dl and 20/ml, respectively, after 1 year of deferasirox treatment is clinically significantly. Due to gradual improvement of anemia, requirement of RBC transfusion had continuously decreased during the study period (R2=0.31). This subanalysis of KAMS0112 study demonstrates that ICT using deferasirox can be effective in improving anemia and thrombocytopenia in the transfusional iron overload patients with AA, as well as reducing serum ferritin level and LIC. Further studies might be required to elucidate the mechanism involved in the improvement of hematopoiesis associated with correction of deranged intracellular iron homeostasis. Disclosures: Min: Novartis: Research Funding. Yoon:Novartis: Research Funding. Kim:Novartis: Research Funding. Lee:Novartis: Research Funding. Lee:Novartis: Research Funding. Won:Novartis: Research Funding. Shim:Novartis: Research Funding. Kim:Novartis: Research Funding. Seung:Novartis: Research Funding. Kim:Novartis: Research Funding. Lee:Novartis: Research Funding. Chung:Novartis: Research Funding. Hyun:Novartis: Research Funding. Jo:Novartis: Research Funding. Jung:Novartis: Research Funding. Sohn:Novartis: Research Funding. Yoon:Novartis: Research Funding. Kim:Novartis: Research Funding. Joo:Novartis: Research Funding. Cheong:Novartis: Research Funding.

Significant Impact Of Iron Chelation After Allogeneic Hematopoetic Stem Cell Transplantation On Disease Recurrence: Potential Anti-Leukemic Activity

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 180-180 ◽  
Author(s):  
Mauricette Michallet ◽  
Mohamad Sobh ◽  
Stephane Morisset ◽  
Helene Labussiere ◽  
Marie Y. Detrait ◽  
...  
Keyword(s):  
Stem Cell ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Disease Recurrence ◽  
Hazard Ratio ◽  
Iron Chelators ◽  
Myeloid Leukemias

Abstract Iron overload (IO), primarily related to multiple red blood cell transfusions, is a relatively common complication in allogeneic hematopoietic stem cell transplant (allo-HSCT) recipients. Elevated pre-transplant ferritin level, a surrogate marker of iron overload, was demonstrated to be an important cause of mortality and morbidity in patients who have undergone allo-HSCT. Excessive iron accumulation results in tissue damage and organ failure, mainly as a result of the generation of free radicals that cause oxidative damage and organ dysfunction. Iron chelators have been widely used leading to normalisation for ferritine level and lower IO-related complications. As iron has a fundamental role in cell survival affecting pathways involved in DNA synthesis, cell differentiation, and apoptosis, some studies evaluated the anti-proliferative activity of iron chelators in cancer and leukemia patients on disease recurrence. The objective of this study was to determine at a first time the impact of serum ferritin level measured at time of allogeneic HSCT in adult patients with hematological disorders on the different outcomes and to investigate at a second time the role of iron chelation on relapse incidence. We included 158 patients, 100 males and 58 females with a median age of 45 years (18-67) who underwent allo-HSCT between 2002 and 2010. There were 83 acute myeloid leukemias, 10 chronic myeloid leukemias, 11 myelodysplastic syndromes, 7 myeloproliferative disorders, 19 myelomas, 9 non-Hodgkin lymphomas, 6 Hodgkin diseases, 5 aplastic anemias and 3 hemoglobinopathies. Sixty-seven (42%) patients were sex mismatched (F→M:37; M→F:30); for ABO compatibility, 61% were compatible, 18% had minor incompatibility and 21% had major incompatibility. Concerning the HSCT procedures, 60 patients (38%) received peripheral blood stem cell and 98 (62%) received bone marrow from 97 (61%) HLA related donors [matched, n=76; mismatched, n=21], and 61 (39%) HLA unrelated donors [matched, n=36; mismatched, n=25] after myeloablative [n=64, (41%)] or reduced intensity conditioning [n=94, (59%)]. At transplantation, 91 (58%) were in complete remission (CR) or chronic phase [CR1: n=61 (67%); ≥CR2: n=30 (33%)]. The median serum ferritin level at HSCT was 1327 microg./l (26-14136); 31(20%) patients had a level 26-500, 33 (21%) had a level 500-2500, and 94 (59%) >2500. There was no significant correlation between the different ferritin levels, disease kind and status at HSCT. After transplantation, 23 patients received iron chelating agents after a serum ferritin level of 1000 microg/l and stopped when the level decreased below 1000. The cumulative incidence of acute GVHD ≥ II at 3 months was 14% (11-16.5) with 10.5% (8-13) for grade III and 7% (5-9) for grade IV; the 1 year cumulative incidence of limited and extensive chronic GVHD were 4% (2-6) and 12.4% (9-16) respectively. After a median follow-up of 18 months (1-106), the 5 years OS probability was 65% for patients with ferritin level below 500 microg./l, 39% for level between 500 and 2500 microg./l and 28% for level > 2500 micog./l, [Hazard ratio= 3.5 (1.5-8.1), p=0.002]; this was explained by a significant higher TRM in patients with level >2500 [Hazard ratio= 4.3 (1.02-18), p=0.04]. Interestingly, we found in multivariate analysis that patients receiving iron chelators had significantly better OS [5 years OS= 59% vs. 34% for non-chelated patients, Hazard ratio= 0.34 (0.15-0.76), p=0.008], (Figure 1a), and experienced less disease relapse [5 years relapse incidence= 18% vs. 41% for non-chelated patients, Hazard ratio= 0.22 (0.07-0.73), p=0.012], (Figure 1b). In conclusion, we confirmed the negative impact of iron overload on the outcomes allo-HSCT recipients. More importantly, we demonstrated that iron chelators have a positive impact in reducing disease relapse by the possible mechanism of iron deprivation in leukemic cells. This clinical observation needs to be confirmed by prospective randomized trials.Figure 1a: Overall survival probability and b: relapse incidence in patients with or without iron chelationFigure 1. a: Overall survival probability and b: relapse incidence in patients with or without iron chelation Disclosures: Michallet: Novartis: Honoraria, Research Funding. Nicolini:Novartis: Consultancy, Honoraria, Research Funding.

Different Adverse Event Profiles and Significant Hematological Improvement Are Noted When Deferasirox Was Used in Adult Patients with Chronic Transfusion-Related Iron Overload in Taiwan: Results from a Prospective Observational Clinical Trial

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4554-4554
Author(s):  
Bor-Sheng Ko ◽  
Ming-Chih Chang ◽  
Tzeon-Jye Chiou ◽  
Te-Kau Chang ◽  
Yeu-Chin Chen ◽  
...  
Keyword(s):  
Adverse Events ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Serum Ferritin Level ◽  
Ferritin Level ◽  
Platelet Response ◽  
Chronic Anemia ◽  
Erythroid Response ◽  
The Mean ◽  
Grade 3

Abstract Background Iron overloading is a common problem for adult with myelodysplastic syndrome (MDS), aplastic anemia (AA) or other chronic anemia. Deferasirox (DFX) has been proven as an effective therapy to chelating iron in these patients. Anyway, the safety of DFX is still a concern, and the information of safety profiles and efficacy are less understood in Taiwan. This study is planned primarily to collect long-term safety data of DFX treatment in iron-overloaded MDS, AA and other chronic anemia patients in Taiwan. Study Design This is an observational, single-arm, and multi-center study. Low-risk MDS or AA patients with transfusion-related iron overload, or patients with other anemia and serum ferritin more than 2000 ug/ml, were enrolled within the 18-month enrolling period if DFX is planned to be prescribed. Exposure of iron chelating agents other than DFX before trial initiation was allowed. The initial dose and subsequent adjustment of DFX were up to investigator¡¦s preference. All patients were followed for 3 years for adverse events (AEs) and disease outcomes. Results From 2009 to 2011, 79 patients were enrolled in this study, including 38 MDS, 23 AA and 18 other chronic anemias. Forty-seven cases (59.5%) were male, with mean age 64.3¡Ó17.8 y/o. Fifty-six (70.9%) subjects failed to complete the 3-year study period, but only 8 (10.1%) of the subjects withdrew DFX due to drug-related AEs. The mean DFX exposure dose during study was 17.7¡Ó4.02 mg/Kg/day. In contrast with those reported in literature, the most frequently reported drug-related AEs were rash (16, 20.3%), diarrhea (11, 14.0%), hypercreatinemia (8, 10.1%), pruritus (7, 8.9%), and so on (as in Table 1). When classified by organ systems, skin disorders were the frequently reported one (26, 32.9%), and followed by GI disorders (n=24, 30.4%). Grade 3-4 drug-related adverse events were rare (n=4, 5.1%). For all subjects, DFX could effectively decrease serum ferritin level from baseline (-985+/-2090 ng/ml (p=0.0154 vs. baseline) and -1710+/-2290 ng/ml (p=0.0424 vs. baseline) at 1 yr and 3 yr, respectively) (as in Figure 1). Notably, after DFX usage, 23 patients (32.4%) developed erythroid response according to IWG 2006 criteria; the mean hemoglobin could increase from 7.77+/-1.63 gm/dl (baseline) to 8.25+/-2.60 gm/dl (at 36 month, p=0.6172 vs. baseline), when the average transfusion amount was decreased from 2.3+/-1.4 units (baseline) to 1.6¡Ó0.5 units (at 36 months, p=0.0406 vs. baseline). (as in Figure 2). Ten patients (10/46, 21.7%) had platelet response. For the 38 MDS patients, DFX also could significantly lower serum ferritin level (-590+/-2490 ng/ml (p=0.4095 vs. baseline) and -1310+/-362 ng/ml (p=0.0013 vs. baseline) at 1 yr and 3 yr, respectively) but seemed to have a less extent than that in overall population. Similarly, 10 patients (21.7%) developed erythroid response after DFX use. The mean hemoglobin increment (from 7.67+/-1.67 gm/dl (baseline) to 8.55+/-3.45 gm/dl (at 36 month, p=0.6012 vs. baseline)) and the decrease of average transfusion amount (from 2.1+/-1.2 units (baseline) to 1.6+/-0.6 units (at 36 months, p=0.2943 vs. baseline) were not significant, probably due to low case number (as in Figure 2). Four (4/19, 21.1%) patients experienced platelet response. Conclusion This study showed that the profiles of AEs regarding DFX use for adult anemic patients with transfusion-related iron overload in Taiwan were significantly different from those reported in Western countries. The AE-related discontinuation rate was also relatively low. An expected efficacy to lowering serum ferritin by DFX, and a significantly degree of hematological improvement was noted, too. Table 1. Drug-related adverse events, for all events with incidences > 5% and all grade 3-4 events: Table 1. Drug-related adverse events, for all events with incidences > 5% and all grade 3-4 events: Figure 1. Figure 1. Figure 2. Figure 2. Disclosures Chang: Novartis: Honoraria.

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