Bone Denisty Change in Children With Beta Thalassemia Major

Assiut University, Pediatrician Department

Bone denisty changes in children with beta thalassemia major

Study Overview

• Status: Not yet recruiting
• Conditions: Evaluate Bone Denisty Changes in Children With Beta Thalassemia Major
• Intervention / Treatment: Radiation: Dexa scan

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,

• Study Type: Observational
• Enrollment (Estimated): 42

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

Children with beta thalassemia major with transfusion dependent

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Controlled beta thalassemia major; Controlled beta thalassemia major Hb>9g/dl , serum ferritin <500ng/ml	Radiation: Dexa scan; No intervention
Uncontrolled beta thalassemia major; Uncontrolled beta thalassemia major Hb<9g/dl , serum ferritin >500ng/ml	Radiation: Dexa scan; No intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate bone denisty in children with beta thalassemia major	To evaluate bone denisty in transfusion dependent beta thalassemia major & its relation to serum minerals &vit D To evaluate bone denisity in transfusion dependent beta thalassemia major & its relation to serum minerals &vit D To evaluate bone denisity in transfusion dependent beta thalassemia major &its relation to serrum minerals &vitD	One year

Collaborators and Investigators

• Sponsor: Marwa Hassan Abdelhamed Hassan
• Collaborators: Assiut University

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Estimated): December 15, 2023
• Primary Completion (Estimated): December 15, 2024
• Study Completion (Estimated): December 31, 2024

Study Registration Dates

• First Submitted: September 29, 2023
• First Submitted That Met QC Criteria: September 29, 2023
• First Posted (Actual): October 5, 2023

Study Record Updates

• Last Update Posted (Actual): October 5, 2023
• Last Update Submitted That Met QC Criteria: September 29, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: Thalassemia major in children

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No