Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Transfusion Dependent Beta Thalassemia, Sickle Cell Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Primary outcome measures
- Quality of Life [Time frame: Past 12 months]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 1 center
- Georgetown University Medical Center — Washington D.C.
Identifiers
NCT: NCT06664541 · STUDY00007772