Back to Search
Start Over
Gene Therapy in Hemophilia: A Transformational Patient Experience.
- Source :
- Journal of Patient Experience; 8/31/2023, p1-10, 10p
- Publication Year :
- 2023
-
Abstract
- Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact. [ABSTRACT FROM AUTHOR]
- Subjects :
- HEMOPHILIA treatment
RESEARCH
LEISURE
SPORTS participation
FOCUS groups
HEALTH services accessibility
PATIENT participation
SELF-perception
PATIENT satisfaction
HEALTH outcome assessment
ACTIVITIES of daily living
PATIENTS' attitudes
EXPERIENCE
PHYSICAL activity
GENE therapy
QUALITY of life
RESEARCH funding
QUESTIONNAIRES
COMMUNICATION
PATIENT-professional relations
EVALUATION
Subjects
Details
- Language :
- English
- ISSN :
- 23743735
- Database :
- Complementary Index
- Journal :
- Journal of Patient Experience
- Publication Type :
- Academic Journal
- Accession number :
- 171338261
- Full Text :
- https://doi.org/10.1177/23743735231193573