Complications in Children With B- Thalassemia Major

Assiut University

Status: Unknown

Conditions: Beta-thalassemia

Treatments: Other: Demographic, physical examination, data will becollected .

Study type: Observational

Funder types: Other

Identifiers

NCT03462784

Prev TM

Details and patient eligibility

About

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.

Full 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.

Enrollment: 201 estimated patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

201 participants in 1 patient group

Cases

Treatment:

Other: Demographic, physical examination, data will becollected .