Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen

Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelators

repeated transfusions are the mainstay of disease management in most patients with transfusion dependent beta thalassemia.iron overload predispose to oxidative stress and tissue injury.

oxidative stress play important role in pathogenesis of anemia in beta thalassemia.

vitamin E is often depleted in thalassemia patients.

Study Overview

• Status: Completed
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Dietary supplement: vitamin E; Other: PLACEBO

Detailed Description

oxidative stress status is very important in thalassemic patients and explains the different manifestations in thalassemic patients.

vitamin E is fat soluble vitamin shown to reduce the oxidative stress in thalassemia and to reduce lipid peroxidation of red cell membranes.

therefore, this study shows the safety of oral vitamin E as adjuvant therapy to three iron chelators : desferoxamine,deferiprone and deferasirox in moderately iron overloaded children and adolescents with transfusion dependent beta thalassemia and its relation to iron overload over one year.

• Study Type: Interventional
• Enrollment (Actual): 180
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Egypt
  • Abbasia
    • Cairo, Abbasia, Egypt, 11517
      • Ain Shams University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Beta thalassemia patients on regular packed red blood cells transfusion,with vitamin E deficiency
• serum ferritin 1000-2500 ug/l
• cardiacT2*>10ms and ejection fraction >55%

Exclusion Criteria:

• Diabetes,active hepatitis(serum transaminases >2 times ULN)
• renal impairment (serum creatinine>2times ULN)
• participation in previous investigational drug study with 3 months preceding screening supplementation with antioxidants or vitamins within 3 months prior to the study and patients with known allergy to vitamin

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Beta thalassemia patiens with oxidative stress and taking iron chelators receiving vitamin E; Beta thalassemia patiens with oxidative stress and taking iron chelators(desferoxamine,deferiprone or deferasirox) receiving vitamin E in dose 400 mg daily for 12 months	Dietary supplement: vitamin E; Vitamin E will be taken for 12 months for beta thalassemia patiens on regular packed red blood cell transfusion and chelators(desferoxamine,deferiprone and deferasirox )
Placebo Comparator: Beta thalassemia patiens with oxidative stress and taking iron chelators receiving placebo; B thalassemia patiens with oxidative stress and taking iron chelators(desferoxamine,deferiprone or deferasirox) receiving placebo for 12 months	Other: PLACEBO; placebo will be taken for 12 months for beta thalassemia patiens on regular packed red blood cell transfusion and chelators(desferoxamine,deferiprone and deferasirox )

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
effect of vitamin E on Liver iron concentration (LIC)	measuring the LIC using MRI (magnetic resonance imaging) in mg/g	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
effect of vitamin E on antioxidants enzymes	measuring antioxidants enzymes; antioxidant enzymes,	12 months
effect of vit E supplementation on SF	measuring serum ferritin(SF)in ng/ml	12 months

Collaborators and Investigators

• Sponsor: Ain Shams University

Publications and helpful links

General Publications

• Fibach E, Rachmilewitz EA. The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:10-6. doi: 10.1111/j.1749-6632.2010.05577.x.

Study record dates

Study Major Dates

• Study Start (Actual): July 5, 2021
• Primary Completion (Actual): September 11, 2022
• Study Completion (Actual): October 11, 2022

Study Registration Dates

• First Submitted: July 6, 2024
• First Submitted That Met QC Criteria: July 17, 2024
• First Posted (Actual): July 19, 2024

Study Record Updates

• Last Update Posted (Actual): July 19, 2024
• Last Update Submitted That Met QC Criteria: July 17, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Oxidative stress; vitamin E; thalasssemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Protective Agents; Micronutrients; Vitamins; Antioxidants; Vitamin E
• Other Study ID Numbers: MD12/2020

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No