Complications in Children With B- Thalassemia Major

NCT ID: NCT03462784

Last Updated: 2020-01-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

201 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-08-01

Study Completion Date

2020-12-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Beta-thalassemia

Study Design

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Observational Model Type

OTHER

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Cases

Demographic, physical examination, data will becollected .

Intervention Type OTHER

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

Interventions

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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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Assiut University

OTHER

Sponsor Role lead

Responsible Party

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Tahany Naeem Saied Matta

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Central Contacts

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Mohamed Hamdy Ghazaly, M.A. Ph.D.

Role: CONTACT

01001296603

Ismail Lotfy Mohamad, M.A. Ph.D.

Role: CONTACT

01063398967

References

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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.

Reference Type RESULT
PMID: 18568278 (View on PubMed)

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.

Reference Type RESULT
PMID: 20233970 (View on PubMed)

Other Identifiers

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Prev TM

Identifier Type: -

Identifier Source: org_study_id

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