Comparative Efficacy and Safety Between Deferiprone and Deferasirox with Special Reference to Serum Ferritin Level and Cardiac Function in Bengali β-Thalassemia Major Children

Abstract Objectives: To evaluate the influence of pretransplantation serum ferritin on children β-thalassemia major (β-TM) undergoing allogeneic hematopoietic stem cell transplantation. Methods: A retrospective analysis of 266 HLA-matched children with β-TM from January 2009 and November 2014 was performed. Transplantation conditioning regimen of these children was the NF-08-TM protocol. Median follow-up time was 28 months (3~62months). We observed the relationship between pretransplantation serum ferritin level and transplantation complications which included infection, graft versus host disease(GVHD),veno-occlusive disease(VOD) and death. Results: Transplantation-related death occurred in 18 of 266 patients (6.8%). Five-year overall survival (OS) was found to be 92.8%. Among various complications, only infection was significantly associated with the high serum ferritin level (t=-2.673, P=0.008), especially serum ferritin above 3449.5μg/L(P=0.000). Meanwhile infection was the most common complication and severe infection would be main cause of deaths. Conclusions: NF-08-TM conditioning regimen was the optimization for HLA-matched β-TM patients. High pretransplantation serum ferritin level would bring high infection occurrence. Disclosures No relevant conflicts of interest to declare.

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Deferiprone effectiveness in thalassemia major children with or without hepatitis B or C virus infection: a non-randomized study

Paediatrica Indonesiana ◽

10.14238/pi50.2.2010.105-12 ◽

2010 ◽

Vol 50 (2) ◽

pp. 105

Author(s):

Yovita Ananta ◽

Pustika Amalia Wahidiyat ◽

Hanifah Oswari

Keyword(s):

Serum Level ◽

Virus Infection ◽

Hepatitis B ◽

Serum Ferritin ◽

Serum Ferritin Level ◽

Ferritin Level ◽

Randomized Study ◽

Thalassemia Major ◽

Transfusion Therapy ◽

Randomized Clinical Study

Background A high incidence rate of hepatitis B or C virusinfection is found among thalassemia children in Indonesia. Thismay influence deferiprone effectiveness.Objective To determine the effectiveness of deferiprone inthalassemia children with or without hepatitis B or C virusinfection.Methods A non-randomized clinical study was performed atThalassemia Center Jakarta. Subjects were thalassemia childrenwith serum ferritin level > 1000 ng/mL who had hepatitis B orC virus infection. A match control pair was recruited based onsimilar duration of transfusion therapy, thalassemia type, andinitial serum ferritin level. All subjects received initial deferipronedose of 50 mg/kg/day for 3 months. Those whose ferritin decreased2:: 10% continued to receive deferiprone of 50 mg/kg/day for thefollowing 3 months. Otherwise, deferiprone dose was adjustedto 75 mg/kg/day.Results Forty-eight subjects were recruited. After 3 months oftreatment, 16/24 subjects without and 6/24 subjects with hepatitisB or C had their ferritin level decreased 2:: 10%. Mean ferritinserum level of all subjects after 6 months was significantly reducedfrom 4734 (SD 2116) to 3695 (SD 1709) ng/mL. Lower meandeferiprone dose, lower mean post- study ferritin serum level andhigher mean percentage of ferritin serum level decrement werefound in subjects without hepatitis B or C infection than thosewith infection.Conclusions Deferiprone 50-7 5 mg/kg/day for 6 months is effectivein reducing serum ferritin level of thalassemia major children; itis more effective in thalassemia children without hepatitis B or Cvirus infection.

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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.

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Left ventricular dysfunction in transfusion dependent beta Thalassemia major patients in Bangladesh

Cardiovascular Journal ◽

10.3329/cardio.v9i1.29539 ◽

2016 ◽

Vol 9 (1) ◽

pp. 31-35

Author(s):

Simu Saha ◽

Tapash Saha ◽

AKM Amirul Morshed ◽

Md Lutful Ehsan Fatmi ◽

Nazneen Umme Zakia ◽

...

Keyword(s):

Diastolic Dysfunction ◽

Serum Ferritin ◽

Cardiac Dysfunction ◽

Serum Ferritin Level ◽

Ventricular Dysfunction ◽

Early Stage ◽

Ferritin Level ◽

Thalassemia Major ◽

Left Ventricular ◽

Beta Thalassemia

Background: Thalassemia major is an inherited haemoglobin disorder resulting in chronic haemolytic anaemia. Patients with beta thalassemia major are maintained on continuous blood transfusion regimens resulting in iron overload that adversely affects both the structure and function of the heart and other vital organs which can be easily prevented with iron chelating therapy. The aim of the study was to detect left ventricular dysfunction at an early stage so that early effective intervention can be done.Methods: A total of 50 patients with beta thalassemia were included in the study by non randomized qualitative purposive sampling from July 2013 to June 2014. Their total body iron status was be assessed by doing serum ferritin level. Left ventricular systolic and diastolic function was assessed by echocardiographyResults: Cardiac dysfunction was present in 11 patients with high incidence in patients with low pre-transfusional haemoglobin group (p=0.4) and in patients having high serum ferritin level (p=0.02). Systolic cardiac dysfunction was present in 7(14%) of patients and diastolic dysfunction was present in 4(8%) of patients. There was a weak but significant correlation between left ventricular ejection fraction and serum ferritin concentration (r=-0.22; p=0.03). Only few (8%) patients had diastolic dysfunction.Conclusion: Patients with beta thalassaemia on an adequate transfusion showed an abnormal left ventricular systolic function. In early stage of disease diastolic function was normal but after repeated transfusion there were impaired relaxation indicating diastolic dysfunction. These findings seem mainly to be related to chronic anaemia and serum ferritin levelCardiovasc. j. 2016; 9(1): 31-35

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Study of serum ferritin level and effect of oral iron chelator in children with b - thalassemia major

International Journal of Clinical Biochemistry and Research ◽

10.18231/j.ijcbr.2020.008 ◽

2020 ◽

Vol 7 (1) ◽

pp. 40-44

Author(s):

Chandrakant Kamariya ◽

◽

Uday N Vachhani ◽

Keyword(s):

Serum Ferritin ◽

Serum Ferritin Level ◽

Ferritin Level ◽

Thalassemia Major ◽

Iron Chelator ◽

Oral Iron ◽

Oral Iron Chelator

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PREVALENCE OF HYPOTHYROIDISM, DELAYED PUBERTY AND DIABETES MELLITUS IN PATIENTS OF Β-THALASSEMIA MAJOR

International Journal of Medical and Biomedical Studies ◽

10.32553/ijmbs.v4i3.1017 ◽

2020 ◽

Vol 4 (3) ◽

Author(s):

Naresh Manne ◽

Bharat Kumar Gupta ◽

Sandeep Kumar Yadav ◽

Saurabh Singhal ◽

Archana Dubey

Keyword(s):

Diabetes Mellitus ◽

Blood Transfusion ◽

Serum Ferritin ◽

Serum Ferritin Level ◽

Ferritin Level ◽

Thalassemia Major ◽

Delayed Puberty ◽

Beta Thalassemia ◽

Endocrine Disorders ◽

Beta Thalassemia Major

Background: Beta-Thalassemia is a genetic disorder which is associated with a lot of complications. Frequent blood transfusions result in increased iron deposition in various tissues leading to dysfunction of many vital organs. Endocrine disorders constitute a major part of such complications increasing the morbidity of thalassemia manifold in the affected patients. Methods: This is a descriptive cross sectional study carried out in 100 diagnosed patients of beta- thalassemia major who had visited the OPD/IPD of Subharti Medical College & affiliated Hospitals, Meerut for routine blood transfusion or for any other complication. Patients were clinically examined and investigated for presence of one or more endocrine disorders on their routine appointments. Results: Endocrine disorders were detected in a total of 82 patients. Diabetes mellitus was detected in 12% patients, hypothyroidism in 36% patients and delayed puberty was found in 72% patients. Mean serum ferritin level was found to be 5831.0±2860.5 ng/ml in beta-thalassemia Major patients, while it was in normal range in control subjects. Conclusion: Research concluded with finding of Delayed puberty (72%), Hypothyroidism (36%) and diabetes mellitus as (12%) in beta thalassemia patients who were on regular blood transfusion therapy. Iron overload as serum ferritin level was found to be highly raised in all study case. On the basis of our study we recommend that early detection and management protocols for these endocrinopathies may improve the life prospects of beta-thalassemia Major patients. Keywords: Endocrine disorders, Hypothyroidism, Delayed puberty, Diabetes Mellitus Serum ferritin, Thalassemia Major.

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