- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03462784
Complications in Children With B- Thalassemia Major
Prevalance and Risk Factors for Complications in Children With B- Thalassemia Major
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.
Study Overview
Status
Conditions
Intervention / Treatment
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
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Mohamed Hamdy Ghazaly, M.A. Ph.D.
- Phone Number: 01001296603
- Email: Ghazally@aun.edu.eg
Study Contact Backup
- Name: Ismail Lotfy Mohamad, M.A. Ph.D.
- Phone Number: 01063398967
- Email: IsmailIbrahim@aun.edu.eg
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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
Study Plan
How is the study designed?
Design Details
- Observational Models: Other
- Time Perspectives: Cross-Sectional
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Cases
|
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 |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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 |
one year
|
Collaborators and Investigators
Sponsor
Publications and helpful links
General Publications
- Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008 Jun;86(6):480-7. doi: 10.2471/blt.06.036673.
- Weatherall DJ. The inherited diseases of hemoglobin are an emerging global health burden. Blood. 2010 Jun 3;115(22):4331-6. doi: 10.1182/blood-2010-01-251348. Epub 2010 Mar 16.
Study record dates
Study Major Dates
Study Start (Anticipated)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Prev TM
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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