Clinical Trials Logo

Clinical Trial Summary

Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world . Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients. Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family. A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes. Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.


Clinical Trial Description

Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world . Thalassemias are genetic diseases characterized by a deficit in the production of hemoglobin, the protein contained in red blood cells that transports oxygen in the bloodstream. The disease is qualified as α-thalassemia or β-thalassemia depending on whether the anomaly results from a defect in the synthesis of the alpha (α) chains or beta (β) chains making up the protein. It is the most prevalent chronic hemolytic anemia in Egypt (85.1%), the carrier rate of thalassemia in 1000 normal subjects was found to be 9-10.2% . Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients. The principles of treatment have been well defined which include (a) maintaining hemoglobin between 9 and 10 g/dl by repeated packed cell transfusion (b) regular chelation therapy to maintain serum ferritin around 1000 ng/dl (c) preventing the development of complications of the disease or secondary to therapy (d) ensuring normal growth and development . This conventional treatment is required for life-long. The main objective of thalassemia life-long treatment is to maintain good quality of life as close to near normal life. there are many Factor that can affect Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family. The assessment of HRQOL in children is essential for the provision of proper care, since it helps in identifying the impact of the disease and treatment on children . A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes. Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests. Previous studies showed significantly different results between patients and controls regarding the intelligence quotient (IQ). ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06058260
Study type Observational
Source Sohag University
Contact Mena G Shawky, resident
Phone 01289496538
Email Menagergeos@med.sohag.edu.eg
Status Not yet recruiting
Phase
Start date October 20, 2023
Completion date October 20, 2024

See also
  Status Clinical Trial Phase
Recruiting NCT04398628 - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed NCT04614779 - Long-term Clinical Study of CN128 in Thalassemia Patients Phase 2
Completed NCT01158794 - Genes Influencing Iron Overload State
Recruiting NCT02995707 - The Effective and Safety of Thalidomide in NTDT Phase 2
Active, not recruiting NCT01935661 - A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT00901199 - Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload Phase 2
Terminated NCT00034528 - Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia Phase 2
Active, not recruiting NCT03655678 - A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia Phase 2/Phase 3
Recruiting NCT05508932 - Atrial Fibrillation in Beta-Thalassemia
Completed NCT03609827 - Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Completed NCT03095326 - Pneumococcal Vaccination for Splenectomised Thalassemia Major Patients in Indonesia Phase 4
Completed NCT03117192 - Zinc Supplementation on Cellular Immunity in Thalassemia Major Phase 4
Completed NCT01443312 - Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia
Completed NCT00744692 - Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders Phase 1
Completed NCT00235391 - Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload Phase 3
Completed NCT05529550 - Assessment of Nutritional Status and Role of Insulin-like Growth Factor-1 in Children
Completed NCT04582110 - The Role of OCTA in Patients Affected by Beta Thalassemia
Recruiting NCT06213402 - RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Recruiting NCT01758042 - Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders N/A