Adherence of Beta Thalssemia Patients to Oral Chelation Therapy

The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. It is now common practice to classify patients as having transfusion dependent β-thalassemia (TDT) or non-transfusion-dependent β-thalassemia (NTDT). Regarding geographical distribution of β-thalassemia, it prevails more in countries on the Mediterranean, South east of Asia and in the east of Europe. In Egypt, it is the most common cause of chronic blood loss: One thousand cases are recorded annually for every 1.5 million live births the disease prevalence is equal to1000 cases per 1.5 million live births (Ghazala et al., 2021).

The only curative treatment for thalassemia currently is a bone marrow transplant. However, it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost. Thus, most patients receive regular blood transfusions accompanied by iron chelation therapy (ICT) as the standard of care. The ideal management of a patient with transfusion-dependent thalassemia (TDT) requires a multidisciplinary therapeutic approach. The main iron chelating agents include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX). Due to poor oral bioavailibility, DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day; DFP and DFX may be administered orally up to three times a day. The known side effects associated with each chelator include infusion reactions in DFO, gastrointestinal distress, agranulocytosis in DFP, and transaminitis in DFP and DFX.

Study Overview

• Status: Recruiting
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Diagnostic test: Serum Ferritin level

• Study Type: Interventional
• Enrollment (Estimated): 200
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Marwa Ali Mousa, resident; Phone Number: 01111815617; Email: marwa.mousaa@med.sohag.edu.eg
• Study Contact Backup: Name: ALzahraa Elsayad Ahmed, professor; Phone Number: 01224340998

Study Locations

• Egypt
  • Sohag, Egypt
    • Recruiting
    • Sohag University Hospital
    • Contact: Magdy M Amin, professor

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. confirmed diagnosis of beta thalassemia major or intermedia,
2. Age between 2-18 years,
3. Receiving regular blood transfusions every 2-5 weeks
4. Prescribed oral iron chelation therapy with deferasirox for at least 1 year prior to enrollment.

Exclusion Criteria:

1. Age less than 2 years and more than 18 years
2. Any cause of blood transfusion other than beta Thalassemia
3. Patients on deferoxamine

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: patient group; Beta thalassemia patients on oral chelation therapy	Diagnostic test: Serum Ferritin level; follow up to the ferritin level in beta thalaseemia patients on oral chelation therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
serum ferritin level in beta thalassemia patients adherent to oral chelation therapy	Adherence of Beta Thalssemia Patients to Oral Chelation Therapy	1 year

Collaborators and Investigators

• Sponsor: Sohag University

Publications and helpful links

General Publications

• Belhoul KM, Bakir ML, Saned MS, Kadhim AM, Musallam KM, Taher AT. Serum ferritin levels and endocrinopathy in medically treated patients with beta thalassemia major. Ann Hematol. 2012 Jul;91(7):1107-14. doi: 10.1007/s00277-012-1412-7. Epub 2012 Jan 28.
• Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. doi: 10.1111/j.1537-2995.2007.01416.x.
• Kannan S, Singh A. Compliance score as a monitoring tool to promote treatment adherence in children with thalassemia major for improved physical growth. Asian J Transfus Sci. 2017 Jul-Dec;11(2):108-114. doi: 10.4103/ajts.AJTS_61_16.
• Nazir HF, Alshizawi M. Neutropenia and Life-threatening Agranulocytosis Among Children With beta-Thalassemia Treated With Oral Iron Chelators in a Community With Background of Ethnic Neutropenia. J Pediatr Hematol Oncol. 2020 Nov;42(8):e750-e755. doi: 10.1097/MPH.0000000000001699.

Study record dates

Study Major Dates

• Study Start (Actual): July 14, 2024
• Primary Completion (Estimated): July 14, 2025
• Study Completion (Estimated): July 14, 2025

Study Registration Dates

• First Submitted: August 21, 2024
• First Submitted That Met QC Criteria: August 21, 2024
• First Posted (Actual): August 23, 2024

Study Record Updates

• Last Update Posted (Actual): August 23, 2024
• Last Update Submitted That Met QC Criteria: August 21, 2024
• Last Verified: August 1, 2024

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: Soh-Med-24-07-17MS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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