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

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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