Clinical trial NCT03462784: Complications in Children With B- Thalassemia Major
Official title: Prevalance and Risk Factors for Complications in Children With B- Thalassemia Major
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Beta-thalassemia
Lead sponsor: Assiut University
Sponsor class: OTHER
First posted: 2018-03-13
Enrollment: 201
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children who diagnosed as B thalassemia major

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.

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.

Eligibility 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

Primary outcomes:
measure: Prevelance of Comlications of Beta -Thalassemia major in children
timeFrame: one year
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
References:
citation: 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.
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.