“Efficacy and Safety of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Children Hospital of Bangladesh”

Background: Thalassemia is an inherited hemoglobin disorder; mostly require life-long blood transfusions, leading to chronic iron overload which causes growth failure, delayed sexual development in adolescents and vital organ dysfunctions. So, children with thalassemia need lifelong iron chelation therapy. Hence, this study conducted with the aim of to evaluate efficacy and safety of Deferasirox in pediatric thalassemia patients. Materials and methods: This is an observational, prospective, single-centered hospital-based study conducted in a tertiary care teaching hospital from June 2018 to December 2019. Total sixty children (age 2-18 years) with beta thalassemia major and hemoglobin E beta thalassemia with iron overload were enrolled to commence deferasirox. Efficacy and safety we observed by measuring serum ferritin three monthly and SGPT, SGOT & serum creatinine monthly. Results: The serum ferritin level of 72% patients reduced significantly after 12 months in comparison to baseline level. There was no serious adverse effect except mild abdominal pain, nausea & vomiting and transaminitis. Conclusion: Deferasirox is efficacious in reducing iron overload of the body when administer at optimum dose over at least one year and presenting a safe as well as convenient alternative for most of the transfusion dependent pediatric thalassemia patients.

Download Full-text

Noninvasive assessment of iron overload by magnetic resonance imaging

Pediatric Hematology/Oncology and Immunopathology ◽

10.24287/1726-1708-2020-19-3-158-163 ◽

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.

Download Full-text

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

International Journal of Research in Pharmaceutical Sciences ◽

10.26452/ijrps.v9ispl1.1392 ◽

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.

Download Full-text

Elevated Serum Ferritin Levels Are Highly Associated with Diabetes Mellitus and Hypothyroidism in Thalassemia Patients

Blood ◽

10.1182/blood.v120.21.5174.5174 ◽

2012 ◽

Vol 120 (21) ◽

pp. 5174-5174

Author(s):

Sasinee Hantrakool ◽

Adisak Tantiworawit ◽

Ekarat Rattarittamrong ◽

Chatree Chai-adisaksopa ◽

Weerasak Nawarawong ◽

...

Keyword(s):

Diabetes Mellitus ◽

Risk Factor ◽

Iron Overload ◽

Serum Ferritin ◽

Serum Ferritin Level ◽

Ferritin Level ◽

Elevated Serum ◽

Major Risk Factor ◽

Maximum Serum ◽

Hemoglobin E

Abstract Abstract 5174 Background: Endocrinopathiesare well recognized serious complications in thalassemia patients with iron overload. Elevated serum ferritin levels reflect severity of iron overload and are associated with relevant clinical outcomes. Increased serum ferritin > 2, 500 μg/dl has been found to predict the development of cardiac function abnormalities but the predicting serum ferritin level for diabetes and hypothyroidism has not been determined. Method: This is a cross sectional and retrospective study to evaluate the correlation between ferritin levels and endocrinopathies (diabetes, hypothyroidism) in thalassemia patients. All thalassemia patients age > 18 years old, during August 2011 and June 2012 were enrolled. The diagnosis and type of thalassemia were reviewed and confirmed. Diabetes and hypothyroidism were diagnosed by fasting blood sugar and thyroid functions test. Serum ferritin was measured at the same period. The medical record was reviewed for age, sex, splenomegaly, history of splenectomy, transfusion requirement, maximum serum ferritin level, mean serum ferritin level and iron chelation history. Result: Among 92 thalassemia patients [35 male (38%) and 57 female (62%)] with a median age of 30. 6 years (range, 18–71). There were 28 (30. 4%) cases of Homozygous β-thalassemia, 45 cases (48. 9%) of β-thalassemia/Hemoglobin E and 18 cases (19. 5%) of Hemoglobin H or AE Bart's disease. Most patients (60. 9%) underwent splenectomy, while only one third of patients (34. 8%) were NTDT (non-transfusion dependent thalassemia) (Table 1). The mean value of random and maximum serum ferritin levels for the whole group were 2, 408 μg/dl (range279–9, 817) and 5, 101 μg/dl (range 279–37, 656), respectively. The prevalence of diabetes mellitus and impaired fasting glucose (IFG) were 9. 8% (9 cases) and 10. 9% (10 cases), respectively. Patients with diabetes had significantly higher mean maximum serum ferritin levels than those with non-diabetes (11, 241 μg/dl vs. 4, 468 μg/dl, (p=0. 0001) (Table 2). The cut-off point of maximum serum ferritin levels > 2, 500 μg/dl was the major risk factor for the development of diabetes complication in this group of patients. Six patients (6. 5%) and 21 patients (22. 8%) had hypothyroidism and subclinical hypothyroidism, respectively. Patients with hypothyroidism had significantly higher mean maximum serum ferritin levels than those with euthyroidism [(7, 638 vs. 4, 117 μg/dl, (p=0. 003)]. From univariate analysis, the cut-off point of maximum serum ferritin levels > 3, 500 μg/dl was the major risk factor associated with hypothyroidism (p=0. 007). Conclusion: Elevated serum ferritin level is a predictor of the development of diabetes mellitus and hypothyroidism in thalassemia patients with iron overload. The maximum serum ferritin levels of greater than 2, 500 and 3, 500 μg/dl are associated with diabetes mellitus and hypothyroidism, respectively. These findings warrant the value of iron chelating therapy to maintain serum ferritin levels below 2, 500 μg/dl to avoid the development of endocrinopathies in patients with thalassemia. Disclosures: No relevant conflicts of interest to declare.

Download Full-text

Correlation between levels of serum ferritin and lipid profile status.

The Professional Medical Journal ◽

10.29309/tpmj/2020.27.12.4254 ◽

2020 ◽

Vol 27 (12) ◽

Author(s):

Safia Rahman ◽

Muhammad Ihtesham Khan ◽

Samiyah Rahman ◽

Saman Waqar

Keyword(s):

Lipid Profile ◽

Serum Ferritin ◽

Serum Cholesterol ◽

Serum Ferritin Level ◽

Ferritin Level ◽

Tertiary Care ◽

Serum Triglyceride ◽

The Body ◽

High Density Lipoproteins ◽

Tertiary Care Centre

Objectives: To analyse the correlation between ferritin level in serum and lipid profile in patients presenting to a Tertiary Care Centre. Study Design: Cross Sectional Descriptive study. Setting: Khyber Teaching Hospital Peshawar. Period: 1st January 2019 to 31st July 2019. Material & Methods: About 60 patients presenting to the laboratory referred for serum lipid profile or serum ferritin were included in the study. Sampling was done by purposive non probability technique. Blood was taken from patients in gel tube. Serum ferritin levels and lipid profile were done in all cases. Lipid profile included serum cholesterol, triglyceride level, low density lipoprotein and high density lipoproteins. The levels were recorded in a proforma. The serum ferritin level was correlated with the lipid profile status in all patient and the results were drawn accordingly. Results: About 60 patients were analysed. The mean age was 20.8± 5.2 years (range: 2 years to 60 years). There were 39 (65%) males and 21(35%) females. Out of 60 cases, serum ferritin level was normal, increased and lower in 15(25%), 40(66.6%) and 5 (8.3%) cases respectively. The cases where serum ferritin was abnormally raised, the significant changes in lipid profile were raised cholesterol and raised triglyceride (in 62.5% and 82.5% cases respectively). Conclusion: There is an association between raised serum ferritin and raised serum cholesterol level and serum triglyceride levels. This supports the link between serum ferritin regulation and lipid metabolism in the body. As dyslipidemia is a major cardiovascular risk factor. Therefore, a raised ferritin level must alert the physician about lipid profile of the patient and should prompt the physician to think about preventive measures against cardiovascular events in order to decrease the morbidity and mortality in patients at risk.

Download Full-text

Estimation of iron overloads using oral exfoliative cytology in beta-thalassemia major patients

CytoJournal ◽

10.4103/1742-6413.178993 ◽

2016 ◽

Vol 13 ◽

pp. 6 ◽

Cited By ~ 1

Author(s):

Swati Leekha ◽

Amit Kumar Nayar ◽

Preeti Bakshi ◽

Aman Sharma ◽

Swati Parhar ◽

...

Keyword(s):

Iron Overload ◽

Prussian Blue ◽

Serum Ferritin ◽

Thalassemia Major ◽

The Body ◽

Transport Cost ◽

Beta Thalassemia ◽

Safe Procedure ◽

Beta Thalassemia Major ◽

Mucosal Cells

Background: Iron overload is a medical condition that occurs when too much of the mineral iron builds up inside the body and produces a toxic reaction. Thalassemia is a genetic disorder of hemoglobin synthesis, which requires regular blood transfusion therapy, and the lack of specific excretory pathways for iron in humans leads to iron overload in the body tissues. It is a major cause of morbidity and mortality in these patients. The estimation of iron levels in exfoliated buccal mucosal cells may provide a simple, noninvasive, and a safe procedure for estimating the iron overload by using the Perls’ Prussian blue stain. Methods: Smears were obtained from buccal mucosa of 40 randomly selected beta-thalassemia major patients and 40 healthy subjects as controls. Smears were stained with Perls’ Prussian blue method. Blood samples were taken for estimation of serum ferritin levels. Images of smears were analyzed using the software image J software version 1.47v and correlated with serum ferritin. Results: Perls’ positivity was observed in 87.5% of thalassemic patients with a positive correlation to serum ferritin levels. Conclusion: The use of exfoliative buccal mucosal cells for the evaluation of iron overloads in the body provides us with a diagnostic medium that is noninvasive, easy to collect, store, and transport, cost effective, and above all reliable.

Download Full-text

Thyroid profile in patients of thalassemia with multiple blood transfusions and high serum ferritin: a cross-sectional study

Pediatric Review International Journal of Pediatric Research ◽

10.17511/ijpr.2020.i08.02 ◽

2020 ◽

Vol 7 (8) ◽

pp. 401-408

Author(s):

Dr. Rohit Khandelwal ◽

◽

Dr. Muralidhar Gundluru ◽

Dr. Leeni Mehta K. ◽

◽

...

Keyword(s):

Serum Ferritin ◽

Thyroid Dysfunction ◽

Ferritin Level ◽

Thalassemia Major ◽

High Serum ◽

The Body ◽

Beta Thalassemia ◽

Blood Transfusions ◽

Beta Thalassemia Major ◽

Tsh Levels

Introduction: Beta-thalassemia major patients undergo regular blood transfusion resulting ingrowth faltering and various endocrine problems including thyroid dysfunction due to iron overloadin the body. This study was conducted to determine the frequency of thyroid dysfunction in childrenpresenting with Beta-thalassemia major on regular blood transfusions. Materials and methods:Sixty children were included with proven beta-thalassemia major who reported to the Department ofPediatrics, VIMS, and RC, Bangalore. Inclusion criteria: 1.Children 4 to 18 years age group .2.Thechild received transfusions for more than 2 years. 3.Children with serum ferritin level >700.Results: In this study, four patients(6.8%) had overt hypothyroidism, eight patients(13.6%) hadsubclinical hypothyroidism and 47 patients(79.7%) had euthyroid status. There was a positivecorrelation between Ferritin and T4, TSH levels. i.e., with an increase in Ferritin level, there was anincrease in T4, TSH levels, and vice versa. However, the correlation was significant with TSH. Therewas a significant negative correlation between Ferritin and T3 levels. i.e with an increase in Ferritinlevel, there was a decrease in T3 levels and vice versa. Conclusion: Thyroid dysfunction can exist inthalassemia patients on multiple transfusions and chelation therapy with high serum ferritin levels.Detection of hypothyroidism is important as inexpensive oral replacement therapy is readilyavailable. Hence regular screening of beta-thalassemia major patients for Serum T3, Serum T4,Serum TSH for early detection and timely treatment could improve the life expectancy and quality oflife of these patients.

Download Full-text

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

Blood ◽

10.1182/blood.v126.23.3368.3368 ◽

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.

Download Full-text

Iron Overload in a Patient with Non-Transfusion-Dependent Hemoglobin H Disease and Borderline Serum Ferritin: Can We Rely on Serum Ferritin for Monitoring in This Group of Patients?

Case Reports in Oncology ◽

10.1159/000507653 ◽

2020 ◽

Vol 13 (2) ◽

pp. 668-673

Author(s):

Mohammad Ali ◽

Mohamed A. Yassin ◽

Maya Aldeeb

Keyword(s):

Iron Overload ◽

Serum Ferritin ◽

Ferritin Level ◽

The Body ◽

Gastrointestinal Absorption ◽

Specific Method ◽

Increased Risk ◽

Endocrine Organs ◽

Secondary Iron Overload ◽

Hemoglobin H Disease

Secondary iron overload is a common complication in the context of hematological diseases, as iron accumulates due to different mechanisms including chronic transfusion, increased gastrointestinal absorption, chronic hemolysis and underlying genetic defects leading to an increase in gastrointestinal absorption of iron. Since the body does not have a mechanism to excrete excess iron, it gets deposited in the heart, endocrine organs, and the liver with the latest being affected less commonly than in primary iron overload disorders like hemochromatosis. Patients with hemoglobin H disease, which is a type of α-thalassemia, are usually transfusion independent, except in occasions where an external stressful factor leads to a drop in hemoglobin and necessitates blood transfusion. Despite this, secondary iron overload is commonly encountered in these patients due to increased gastrointestinal absorption of iron. To avoid the complications associated with iron overload, these patients are usually monitored with serum ferritin, which is an inexpensive widely available method to monitor iron overload. MRI of the liver (Ferriscan) is a more sensitive and specific method to monitor these patients and avoid the long-lasting and sometimes irreversible effect of secondary iron overload. Here we present an interesting case of a patient with hemoglobin H disease, who was monitored with serum ferritin. She had a serum ferritin level considered as a borderline risk for morbidities secondary to iron overload, and an MRI of her liver (Ferriscan) showed significant iron deposition in the liver associated with increased risk of complications secondary to iron overload.

Download Full-text

Serum ferritin levels and it’s correlation with cardiac iron overload with the help of cardiac T2* magnetic resonance imaging

International Journal of Contemporary Pediatrics ◽

10.18203/2349-3291.ijcp20212881 ◽

2021 ◽

Vol 8 (8) ◽

pp. 1374

Author(s):

Shailaja V. Mane ◽

Sharad Agarkhedkar ◽

Dyaneshwar Upase ◽

Tushar Kalekar ◽

P. Sindhura

Keyword(s):

Magnetic Resonance Imaging ◽

Magnetic Resonance ◽

Iron Overload ◽

Serum Ferritin ◽

Ferritin Level ◽

Thalassemia Major ◽

Beta Thalassemia ◽

Resonance Imaging ◽

Mortality And Morbidity ◽

T2 Mri

Background: Frequent blood transfusions in thalassemia major is associated with iron overload in these patients. To reduce the mortality and morbidity, proper usage of iron chelators is necessary to treat iron overload. Cardiac magnetic resonance imaging (MRI) guides in quantification of iron overload in heart. The purpose of this study is to see the correlation between serum ferritin level and T2* MRI in patients with beta thalassemia major.Methods: Period of the study is September 2018 to September 2020. Total 25 patients diagnosed with β-thalassemia major above 5 years of age were enrolled in the study. They were on regular transfusions. Cardiac T2* MRI was done in these patients and correlated with serum ferritin levels.Results: There was no significant correlation observed between cardiac T2* MRI and serum ferritin values (p=0.66, r=-0.094).Conclusions: Our results showed no significant correlation between serum ferritin and cardiac T2* MRI values. Ferritin alone cannot be used as index of myocardial iron overload in thalassemia major.

Download Full-text