Complications in Children With B- Thalassemia Major
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| ClinicalTrials.gov Identifier: NCT03462784 |
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Recruitment Status : Unknown
Verified January 2020 by Tahany Naeem Saied Matta, Assiut University.
Recruitment status was: Not yet recruiting
First Posted : March 13, 2018
Last Update Posted : January 18, 2020
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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 . |
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 : | August 1, 2020 |
| Estimated Primary Completion Date : | October 1, 2020 |
| Estimated Study Completion Date : | December 1, 2020 |
| 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 |
- 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
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| 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 |
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
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
| 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 |
| Responsible Party: | Tahany Naeem Saied Matta, Principal Investigator, Assiut University |
| ClinicalTrials.gov Identifier: | NCT03462784 |
| Other Study ID Numbers: |
Prev TM |
| First Posted: | March 13, 2018 Key Record Dates |
| Last Update Posted: | January 18, 2020 |
| Last Verified: | January 2020 |
| 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 |
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Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |