Sickle Cell Disease, Transfusion Dependent Beta Thalassemia
Conditions
Keywords
quality of life
Brief summary
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness, and to assess the likely impact of gene therapy on patients' quality of life.
Detailed description
The overarching aim of this research is to determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia toward gene therapy to treat their or their child's disease, and to assess the likely impact of gene therapy on patients' quality of life. We will compare and contrast these findings to those obtained from a sample of adult patients and parents of pediatric patients with sickle cell disease. We will also assess perceptions of the pros/cons of gene therapy, including its financial and time costs.
Interventions
None listed
Sponsors
Georgetown University
Study design
Observational model
CASE_ONLY
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* adult patients (ages 18+) or parents of pediatric patients (\<age 18) eligible to receive treatment for their disease with gene therapy
Exclusion criteria
* Non-English-speaking
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Quality of Life | Past 12 months | Health related quality of life is measured by a modified patient reported outcome measure, the Transfusion-dependent Quality of Life (TranQoL) assessment. |
Countries
United States
Outcome results
None listed