Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Transfusion Dependent Beta Thalassemia
- Sponsor
- Georgetown University
- Enrollment
- 100
- Locations
- 1
- Primary Endpoint
- Quality of Life
- Status
- Enrolling By Invitation
- Last Updated
- last year
Overview
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.
Investigators
Eligibility Criteria
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
Outcomes
Primary Outcomes
Quality of Life
Time Frame: 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.