Brief Title
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
Official Title
Prospective Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH)
Brief 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
Detailed 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
Study Type
Observational
Primary Outcome
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
Condition
Thalassemia Major
Intervention
serum ferritin and AST
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Diagnostic Test
Estimated Enrollment
50
Start Date
July 2, 2022
Completion Date
March 2, 2024
Primary Completion Date
December 2, 2023
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Children not treated by chelating agents. Children with stem cell transplantation .
Children with thalassemia minor or intermedia.
Gender
All
Ages
1 Year - 16 Years
Contacts
, +201097602949, [email protected]
Administrative Informations
NCT ID
NCT05303506
Organization ID
thalassemia in AUCH
Responsible Party
Principal Investigator
Study Sponsor
Assiut University
Study Sponsor
, ,
Verification Date
March 2022