Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia

Authoring Organization

Publication Month/Year

October 27, 2023

Last Updated Month/Year

October 30, 2023

Document Type


Country of Publication


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



Diseases/Conditions (MeSH)

D006467 - Hemophilia A, D002836 - Hemophilia B, D015316 - Genetic Therapy


hemophilia, gene therapy, Hemophilia Treatment Center