ClinicalTrials.gov
ClinicalTrials.gov Menu

Complications in Children With B- Thalassemia Major

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03462784
Recruitment Status : Not yet recruiting
First Posted : March 13, 2018
Last Update Posted : June 28, 2018
Sponsor:
Information provided by (Responsible Party):
Tahany Naeem Saied Matta, Assiut University

Brief Summary:

Worldwide, there are more than 60,000 births annually of serious forms of thalassemia .The World Health Organization considers thalassemia to be a major health burden.

Beta- thalassemia is a group of recessively inherited disorders of hemoglobin synthesis characterized by reduced synthesis of the ß-globin chain caused by a mutation. The homozygous state results in severe anemia which needs regular blood transfusion.


Condition or disease Intervention/treatment
Beta-thalassemia Other: Demographic, physical examination, data will becollected .

Detailed Description:

Thalassemia is the most common monogenic disorder in the world .Thalassemia major (beta-thalassemia) affects a significant segment of the population in certain areas of the world. Alterations in migration patterns have changed the geographic distribution of this disease and made it a worldwide health problem with a high frequency in Africa, India, Southeast Asia and the Mediterranean area.

The combination of transfusion and chelation therapy has dramatically extended the life expectancy of these patients, thus transforming thalassemia from a rapidly fatal disease of childhood to a chronic illness compatible with a prolonged life. On the other hand, frequent blood transfusions leading to iron overload and the chronic nature of the disease have contributed to a whole new spectrum of complications in adolescents and young adults suffering from thalassemia major.

Complications associated with beta thalassemia, aside from the aforementioned anemia, are as follows

  • Extramedullary hematopoiesis
  • Asplenia secondary to splenectomy
  • Medical complications from long-term transfusional therapy - Iron overload and transfusion-associated infections (eg, hepatitis) Increased risk for infections resulting from asplenia (eg, encapsulated organisms such as pneumococcus) or from iron overload (eg, Yersinia species))
  • Cholelithiasis (eg, bilirubin stones) Study of the prevelance and risk fators responsible for thalassemia complication can help in adopting appropriating strategies for management of these complication.

Study Type : Observational
Estimated Enrollment : 201 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Official Title: Prevalance and Risk Factors for Complications in Children With B- Thalassemia Major
Estimated Study Start Date : January 1, 2019
Estimated Primary Completion Date : March 1, 2019
Estimated Study Completion Date : October 1, 2019


Group/Cohort Intervention/treatment
Cases Other: Demographic, physical examination, data will becollected .

Demographic data collected includ clinical diagnosis, sex, race, genotype, and phenotype.

Historical data included information regarding immunizations, infection exposures, surgical procedures, organ dysfunction(Organ dysfunction was defined as a history of requiring medical treatment for heart disease, diabetes (Type 1 or 2), hypoparathyroidism, hypothyroidism, growth hormone deficiency, or gonadal failure) age at transfusion initiation, chelation therapy, type of RBC product, transfusion reactions, and antigen matching. Data will be collected from chart review, patient interviews, blood banking records, and laboratory





Primary Outcome Measures :
  1. Prevelance of Comlications of Beta -Thalassemia major in children [ Time Frame: one year ]

    Demographic data collected includ clinical diagnosis, sex, race, genotype, and phenotype.

    Historical data included information regarding immunizations, infection exposures, surgical procedures, organ dysfunction (Organ dysfunction was defined as a history of requiring medical treatment for heart disease, diabetes (Type 1 or 2), hypoparathyroidism, hypothyroidism, growth hormone deficiency, or gonadal failure) age at transfusion initiation, chelation therapy, type of RBC product, transfusion reactions, and antigen matching. Data will be collected from chart review, patient interviews, blood banking records, and laboratory testing. All these data will be collected to know the type of complication and measure the prevelance of these complication




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children who diagnosed as B thalassemia major
Criteria

Inclusion Criteria

  • All patient from 1 year up to 18 year (1year-18 year) admitted at Haematology unit at Assuit University Children Hospital who were diagnosed as Beta thalassemia major

Exclusion Criteria:

  • Age less than 1 year( 1 ≤ year)
  • Children who diagnosed as other types of thalassemia except Beta thalassemia major

Information from the National Library of Medicine

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): NCT03462784


Contacts
Contact: Mohamed Hamdy Ghazaly, M.A. Ph.D. 01001296603 Ghazally@aun.edu.eg
Contact: Ismail Lotfy Mohamad, M.A. Ph.D. 01063398967 IsmailIbrahim@aun.edu.eg

Sponsors and Collaborators
Assiut University

Publications of Results:
Responsible Party: Tahany Naeem Saied Matta, Principal Investigator, Assiut University
ClinicalTrials.gov Identifier: NCT03462784     History of Changes
Other Study ID Numbers: Prev TM
First Posted: March 13, 2018    Key Record Dates
Last Update Posted: June 28, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn