Long Term Outcomes in β Thalassemia Major

NCT02307786 | Completed

• 1 other identifier: SCT 0414B
• Study Type: observational
• Target: 176
• Locations: 1 country
• Sites: 1

Brief Summary

Beta thalassemia (β-thalassemia) is the most common genetic disease worldwide. Individuals with thalassemia are born with a defect in hemoglobin. Hemoglobin is a protein in red blood cells that carries oxygen to vital organs such as the brain, heart, lungs and kidneys. Thalassemia major is a hereditary anemia characterized by little or no ß-globin production, which results in hemolysis (breakdown or destruction of red blood cells) due to the formation of unstable alpha-globin tetramers and ineffective erythropoiesis which is uniformly fatal in the absence of regular transfusions. Although improvements in conservative treatment have improved the prognosis of thalassemia considerably disease and transfusion related complications in affected patients progress over time, causing severe morbidity and shortened life expectancy. Substantial lifelong health care expenses are also involved, often a financial burden for families and unsustainable in most developing countries. The hypothesis is that patients who had beta thalassemia who have undergone a hematopoietic stem cell transplant (HSCT) and are \>1 year post-HSCT will have less long term comorbidities and a higher quality of life (QOL) as compared to those with beta thalassemia who are maintained on supportive care. In order to assess quality of life, a quality of life questionnaire will be asked. Extraction of data from the patient's medical record will also be used to determine any comorbidities that have occurred after either a HSCT or supportive care therapy.

Conditions

Thalassemia Major

Outcome Measures

Primary Outcomes (1)

• Beta-Thalassemia patients and Quality of Life after HSCT versus supportive care therapy

  5 years

Study Arms (2)

Beta-Thalassemiall -Transplantation

Beta-Thalassemia Supportive Care

Eligibility Criteria

• Age: 0 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population will be divided into the following groups: * Recipients that are \>1 year post allogeneic HSCT from any donor * Patients ('Controls') enrolled on the Thalassemia Longitudinal Cohort study and/or the Thalassemia Clinical Registry Network (TCRN) and continue to receive supportive care

You may not qualify if:

• \<1 year post-allogeneic HSCT for Beta-Thalassemia
• Patient expired prior to 1 year post-HSCT
• Autologous stem cell transplantation for Beta-Thalassemia

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

Study Sites (1)

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2014
• First Posted: December 4, 2014
• Study Start: June 1, 2014
• Primary Completion: November 13, 2015
• Study Completion: November 30, 2016
• Last Updated: February 11, 2025

Record last verified: 2025-02

Locations

US (1)