Bone Denisty Change in Children With Beta Thalassemia Major

NCT ID: NCT06069089

Last Updated: 2023-10-05

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 42 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-12-15
• Study Completion Date: 2024-12-31

Brief Summary

Bone denisty changes in children with beta thalassemia major

Detailed Description

Beta Thalassemia major (TM) is a hereditary disease caused by defective Beta globin chain synthesis, resulting in abnormal as well as a decreased quantity of globin chains, ineffective erythropoiesis, haemolysis and increased red blood cell turnover (Cooley, etal, 1927). described the first patient with anemia, splenomegaly, cranial & facial bone enlargement. Pathophysiology of bone denisity changes in beta thalassemia major Several studies had been previously evaluated; shown that multiple factors may act in concert to produce bone disease in beta thalassemia major (TM) including bone marrow expansion (Shamshirsaz, etal, 2003). hypogonadism (Anapliotou,Saka&Jensen,1998), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman,etal,1998), altered pattern of cytokines (Morabito,etal,2007), iron deposit in bone ((Bordat,etal,1993),deferoxamine bone toxicity (Chan ,etal, 2002),and vitamin D deficiency (Dandona, etal, 1987). Some of these pathogenic factors, directly and/or indirectly, affect osteoblastic population, leading to depressed bone formation, while others often increase osteoclastic bone resorption.

Complications of transfusion dependent poorly controlled beta thalassemia major are;(1)-Osteoprosis; Iron overload impairs osteoid maturation and inhibits local mineralization to form focal osteomalacia. In addition, integration of iron in calcium hydroxyapatite affects the growth of crystals, which causes mineralization failure (Chan, etal, 2002), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman, etal, 1998),altered pattern of cytokines (Morabito,etal, 2007), iron deposit in bone (Bordat, etal, 1993), deferoxamine bone toxicity (Chan, etal,2002). and vitamin D deficiency (Dandona, etal, 1987). (2)-Fractures; The introduction of red blood cell transfusion and concomitant iron chelation therapy has led to improved bone health through various mechanisms. It leads to a reduction in medullary expansion and cortical bone thinning, the reduced incidence of hypogonadism, and a reduction in other endocrine complications such as hypoparathyroidism and metabolic disorders that predispose to low bone density and fractures( Multicentre study, italian working group 1995). Z-score of bone density will be calculated. Z score is the preferred parameter in children. which is calculated as the number of standard deviations above or below the mean for the patient's age, sex,

Conditions

Evaluate Bone Denisty Changes in Children With Beta Thalassemia Major

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: CROSS_SECTIONAL

Study Groups

Controlled beta thalassemia major

Controlled beta thalassemia major Hb\>9g/dl , serum ferritin \<500ng/ml

Dexa scan

Intervention Type: RADIATION

No intervention

Uncontrolled beta thalassemia major

Uncontrolled beta thalassemia major Hb\<9g/dl , serum ferritin \>500ng/ml

Dexa scan

Intervention Type: RADIATION

No intervention

Interventions

Dexa scan

No intervention

Intervention Type: RADIATION

Eligibility Criteria

Inclusion Criteria

• Patient diagnosed as B thalassemia major of both sexes, age range from 10-18 year, who are poorly controlled on frequant blood transfusion. The patient who doesn't have Hb level from 9-10 g/dl, in almost always less than 9 g/dl.

Exclusion Criteria

• Known metabolic bone disease. Less than 10 year or more than 18 year. Bone disease Other than hemolytic anemia.

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assiut University

OTHER

Sponsor Role: collaborator

Marwa Hassan Abdelhamed Hassan

OTHER

Sponsor Role: lead

Responsible Party

Marwa Hassan Abdelhamed Hassan

Assiut university , pediatrician department

Responsibility Role: SPONSOR_INVESTIGATOR

References

Gaudio A, Morabito N, Catalano A, Rapisarda R, Xourafa A, Lasco A. Pathogenesis of Thalassemia Major-associated Osteoporosis: A Review with Insights from Clinical Experience. J Clin Res Pediatr Endocrinol. 2019 May 28;11(2):110-117. doi: 10.4274/jcrpe.galenos.2018.2018.0074. Epub 2018 Jul 11.

Reference Type: BACKGROUND

PMID: 29991466 (View on PubMed)

Other Identifiers

Thalassemia major in children

Identifier Type: -

Identifier Source: org_study_id