Complications in Children With B- Thalassemia Major

Beta-thalassemia Clinical Trial

Official title:

Prevalance and Risk Factors for Complications in Children With B- Thalassemia Major

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03462784
• Other study ID #: Prev TM
• Status: Not yet recruiting
• Start date: August 1, 2020
• Est. completion date: December 1, 2020

Study information

• Verified date: January 2020
• Source: Assiut University
• Contact: Mohamed Hamdy Ghazaly, M.A. Ph.D.
• Phone: 01001296603
• Email: Ghazally[@]aun.edu.eg
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 201
• Est. completion date: December 1, 2020
• Est. primary completion date: October 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Beta-thalassemia
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Assiut University

References & Publications (2)

Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008 Jun;86(6):480-7. — View Citation

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. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevelance of Comlications of Beta -Thalassemia major in children	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