Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

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.

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.

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