Gene Therapy, Hemophilia B
Conditions
Brief summary
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low. Transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. Current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. In addition, few educational resources to explain basic gene therapy concepts exist in languages other than English. This study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries. Primary Objective: To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally Secondary Objectives: 1. To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally 2. To explore healthcare workers beliefs and attitudes about gene therapy 3. To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.
Detailed description
This prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia B (≤2% FIX activity only), caregivers/parents and healthcare workers who care for them at St. Jude and other global countries. Participants will be interviewed virtually using an online video-conferencing platform, phone or in person. Interviews will be performed by experienced qualitative interviewers in the official language of each participating site. Interviews will last approximately 45-60 minutes and will be performed only once. All interviews will be audio recorded and obtained information will be analyzed using semantic content analysis to identify common themes.
Interventions
BEHAVIORALInterview
Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.
Sponsors
St. Jude Children's Research Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Inclusion criteria
* Patients ≥12 years of age * Diagnosis of moderate (FIX ≥1% and ≤2%) or severe (\<1%) hemophilia B * Parents or caregivers to patients with hemophilia 12-17 years of age Inclusion Criteria - Healthcare worker: \- Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients
Exclusion criteria
* Diagnosis of Hemophilia A * Diagnosis of other non-Hemophilia B bleeding disorders
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B | Day 1, or at a future visit (up to approximately 1 year) | Semi-structured interviews will be performed in patients with hemophilia B, their caregivers and healthcare workers to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B including concerns, expectations, and best way to receive information about gene therapy. Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes. |
Countries
Nepal, Peru, Sri Lanka, Thailand, United States, Vietnam
Outcome results
None listed