Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)

Thalassemia Major Clinical Trial

Official title: Prospective Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH)

Status Not yet recruiting

Clinical Trial Summary

To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient

Clinical Trial Description

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 ;

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia Major

• NCT number: NCT05303506
• Study type: Observational
• Source: Assiut University
• Contact: Mohamed Elmostafa Mohamed Hemida, master degree
• Phone: +201097602949
• Email: mhemida92@gmail.com
• Status: Not yet recruiting
• Start date: July 2, 2022
• Completion date: March 2, 2024