Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study
- Conditions
- Thalassemia
- Registration Number
- NCT00661804
- Lead Sponsor
- Carelon Research
- Brief Summary
Thalassemias are inherited blood disorders that can cause anemia and other health problems. The goal of this study is to collect information on complications of the disease among people who currently have or previously had thalassemia.
- Detailed Description
Thalassemias are inherited blood disorders that are characterized by low levels of hemoglobin and healthy red blood cells. The two major types of thalassemia are alpha thalassemia and beta thalassemia, and there are several forms of each type. Symptoms can range from mild to severe and may include anemia, delayed growth, bone problems, and an enlarged spleen. People with mild forms of the disease may not need any treatment, while people with moderate to severe thalassemia may be treated with blood transfusions to refresh the healthy red blood cell supply, iron chelation therapy to remove excess iron from the body, and folic acid supplements to help build healthy red blood cells. Stem cell transplants can cure the disease, but they are not widely used because of the difficulty of finding donors. This study will establish a database of people with thalassemia and people who used to have thalassemia to examine the prevalence and incidence of complications related to the disease. Participants' DNA will be analyzed and plasma will be collected for use in future studies. Participants in this study may also be asked if they are interested in enrolling in other Thalassemia Clinical Research Network studies.
This study has enrolled people with thalassemia or people whose thalassemia was cured after undergoing a stem cell transplant. At a baseline study visit, participants with thalassemia will undergo a medical history interview; a medical record review; blood collection; and questionnaires on quality of life, nutritional status, and medication adherence. Follow-up visits will occur once a year for at least 3 years or for the duration of the study and will include repeat baseline testing. Participants who have undergone a successful stem cell transplant will attend only one study visit that will include a medical history interview, a medical record review, and quality of life questionnaires.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 416
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The prevalence and incidence of complications specific to thalassemia and its treatment among participants Measured throughout the duration of the study
- Secondary Outcome Measures
Name Time Method Relationships among adherence, quality of life, and complications of thalassemia Measured throughout the duration of the study Fertility and pregnancy outcomes; causes of mortality and changes in mortality risk; genotypic and phenotypic variation; and body iron burden Measured throughout the duration of the study
Trial Locations
- Locations (14)
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospital of Oakland
🇺🇸Oakland, California, United States
Stanford Hospital
🇺🇸Stanford, California, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Children's Memorial Hospital Chicago
🇺🇸Chicago, Illinois, United States
Children's Hospital Boston
🇺🇸Boston, Massachusetts, United States
Weill Medical College of Cornell University
🇺🇸New York, New York, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Southwestern Medical Center at Dallas
🇺🇸Dallas, Texas, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Scroll for more (4 remaining)Children's Hospital of Los Angeles🇺🇸Los Angeles, California, United States