Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
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|ClinicalTrials.gov Identifier: NCT05303506|
Recruitment Status : Not yet recruiting
First Posted : March 31, 2022
Last Update Posted : March 31, 2022
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|Condition or disease||Intervention/treatment|
|Thalassemia Major||Diagnostic Test: serum ferritin and AST|
Thalassemia major is a heterogeneous disease presenting during infancy or early childhood. Although thalassemia is preventable by premarital counseling and prenatal testing, a large number of children are born with thalassemia, and curative treatment in the form of bone marrow or stem cell transplantation is not possible for the majority of these patients. Such patients need regular transfusions of packed red blood cells (PRBCs) Excess iron can potentially penetrate cells in the liver, heart, endocrine glands and other organs, Iron overload in the heart and liver is known to be a leading cause of morbidity and mortality among patients with transfusion-dependent b-thalassemia major Physical growth is affected in a large number of the patients with transfusion-dependent thalassemia. A study of patients aged 10-27 years with thalassemia major found short stature in 70% of the males and in 73% of the females, thalassemic patients are short, have low rate of growth and BMI and have either delayed or absent pubertal spurt, which is related to low hemoglobin and high ferritin levels and sub-optimal iron chelation therapy. (Najafipour, et al 2008 ).
Therefore, iron-chelating agents, such as deferoxamine, have been used since 1970 and has shown to be effective in chelating iron from the heart and liver, with preservation of heart function and reversal of hepatic fibrosis, respectively Recent advances in the medical management of regular blood transfusion and chelation therapy have allowed most of these patients to have improved survival well into adult life and improved quality of life in patients with severe b-thalassemia promote normal growth, allow normal physical activities, minimize transfusional iron accumulation, adequately suppress bone marrow activity and reduce cardiac overload due to chronic anemia .
Hemoglobin levels maintained at 9-10 g/dl are thought of as optimal , A higher target pre-transfusion hemoglobin level of 11-12 g/dl may be appropriate for patients with heart disease or other medical conditions There is a clear link between the overall iron burden and global toxicity; in patients with thalassemia the liver iron concentration correlates with the total body iron load Serum ferritin and liver iron concentration are widely used to detect iron overload, and the latter can be measured by magnetic resonance imaging (MRI), or liver biopsy, Evidence indicates that serum ferritin is not an accurate measure iron overload and that direct measurement of liver iron concentration via liver biopsy or MRI is more precise
|Study Type :||Observational|
|Estimated Enrollment :||50 participants|
|Official Title:||Prospective Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH)|
|Estimated Study Start Date :||July 2, 2022|
|Estimated Primary Completion Date :||December 2, 2023|
|Estimated Study Completion Date :||March 2, 2024|
- Diagnostic Test: serum ferritin and AST
- Measuring the Effect of different risk factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH) and how these risk factors affect the quality of life by measuring anthropocentric measures [ Time Frame: 2 years ]
Data of the patients will be collected from thalassemic patients in form of
- Personal history (Name, Age ,Sex)
- Therapeutic history (Age of diagnosis , Age of 1st chelator ,Chelator type , Chelator dose , Compliance to treatment )
- Growth parameters (Weight , Height,and mid arm circumference))
- Decreasing the morbidity and mortality and improving the quality of life for thalassemic patient [ Time Frame: 6 months ]At the end of the study there will be recommendations to help in decreasing the morbidity and mortality and improving the quality of life for thalassemic patient
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|Ages Eligible for Study:||1 Year to 16 Years (Child)|
|Sexes Eligible for Study:||All|
|Sampling Method:||Non-Probability Sample|
- All children attending haematology unit in Assiut University Children Hospital with thalassemia treated by iron chelating agents and aged from one year to 16 years old.
- Children not treated by chelating agents. Children with stem cell transplantation . Children with thalassemia minor or intermedia.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05303506
|Contact: Mohamed Elmostafa Mohamed Hemida, master firstname.lastname@example.org|
|Responsible Party:||Mohamed Elmostafa Mohamed Hemida, email@example.com (resident physician of pediatrics), Assiut University|
|Other Study ID Numbers:||
thalassemia in AUCH
|First Posted:||March 31, 2022 Key Record Dates|
|Last Update Posted:||March 31, 2022|
|Last Verified:||March 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn