Adherence of Beta Thalssemia Patients to Oral Chelation Therapy

NCT06568926 | Recruiting

• 1 other identifier: Soh-Med-24-07-17MS
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (1)

• serum ferritin level in beta thalassemia patients adherent to oral chelation therapy

  Adherence of Beta Thalssemia Patients to Oral Chelation Therapy

  1 year

Study Arms (1)

patient group

EXPERIMENTAL

Beta thalassemia patients on oral chelation therapy

Diagnostic Test: Serum Ferritin level

Interventions

Serum Ferritin level DIAGNOSTIC_TEST

follow up to the ferritin level in beta thalaseemia patients on oral chelation therapy

patient group

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Sohag University: lead

Study Sites (1)

Sohag university Hospital

Sohag, Egypt

RECRUITING

Related Publications (4)

• 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.

  PMID: 22281991 BACKGROUND

• 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.

  PMID: 17880620 BACKGROUND

• 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.

  PMID: 28970676 BACKGROUND

• 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.

  PMID: 31876777 BACKGROUND

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Marwa Ali Mousa, resident

CONTACT

01111815617; marwa.mousaa@med.sohag.edu.eg

ALzahraa Elsayad Ahmed, professor

CONTACT

01224340998

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Resident-pediatric department-sohag hospital university

Study Record Dates

• First Submitted: August 21, 2024
• First Posted: August 23, 2024
• Study Start: July 14, 2024
• Primary Completion: July 14, 2025
• Study Completion: July 14, 2025
• Last Updated: August 23, 2024

Record last verified: 2024-08

Locations

EG (1)