Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

St. Jude Children's Research Hospital150 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

150

Conditions

Gene Therapy

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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 Exclusion Criteria - Healthcare worker: * Health care workers who do not participate in the care of hemophilia B patients * Healthcare worker who is conducting the interviews

Locations (6)

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Civil Service Hospital

Kathmandu, Nepal

Hospital Dos De Mayo

Lima, Peru

National Hospital of Sri Lanka

Colombo, Sri Lanka

Ramathibodi Hospital

Bangkok, Thailand

National Institute of Hematology and Blood Transfusion

Hanoi, Vietnam

Outcomes

Primary Outcomes

Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B

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.

Time frame: Day 1, or at a future visit (up to approximately 1 year)