Title
Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia
Authoring Organization
Publication Month/Year
October 26, 2023
Last Updated Month/Year
December 15, 2023
Supplemental Implementation Tools
Document Type
Guideline
Country of Publication
US
Document Objectives
Successful gene therapy will require broad-based, multi-stakeholder participation to define patient eligibility, educate health care providers, educate persons with hemophilia (PWH) and families, and develop shared decision-making tools to facilitate discussions between providers and PWH considering gene therapy. In addition, multi-stakeholder preparation is required to develop clinical protocols for eligibility screening, administration, and follow-up of gene therapy, and to identify ways to remove barriers to access and achieve excellence in clinical delivery to maximize health outcomes. This guideline is focused on hemophilia treatment center (HTC) preparedness for commercial gene therapies for hemophilia.
Inclusion Criteria
Male, Female, Adolescent, Adult, Child, Older adult
Health Care Settings
Ambulatory, Outpatient
Intended Users
Nurse, nurse practitioner, physician, physician assistant
Scope
Management
Diseases/Conditions (MeSH)
D006467 - Hemophilia A, D002836 - Hemophilia B, D015316 - Genetic Therapy
Keywords
hemophilia, gene therapy, Hemophilia Treatment Center