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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0526 - Current challenges in management and implementation of Hemophilia Gene Therapy (HGT) in Spain and Portugal: a survey-based study

Monday, June 24, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall
Background: Hemophilia gene therapy (HGT) is a challenging process for Hemophilia Treatment Centres (HTCs). It is mandatory to adapt infrastructure and human resources and HCTs must organize the clinical process of the people with hemophilia (PwH) candidates to gene therapy.

Aims: A survey-based study was conducted to determine HTCs´ readiness in order to estimate whether the HTCs of Spain and Portugal are ready to provide gene therapy to PwH. Questionnaire includes statements to identify requirements, human resources, availability of gene therapy infusion and capability of implementation of a “hub and spoke” model. (figure 1)

Methods: The topics and statements were validated by a advisory committee composed by 3 hematologist members of the Sociedad Española de Trombosis y Hemostasia. (figure 1) The survey yielded 22 statements across seven topics: 1. willingness to use HGT, 2. capacity to implement advanced cell therapies, 3. capacity to implement gene therapy, 4. ability to discuss with the PwH about the expectations of HGT, 5. laboratory surveillance, 6. patient information resources and 7. informed consents. (table 1)

Results: Survey involved 53 hematologists from 51 centers, with 44 (37 Spanish and 7 Portuguese) hematologist providing willingness to be involved in administration of gene therapy to PwH. (figure 1) This group of 44 hematologists interested in HTG provided valid responses. The main answers of the hematologist are shown in table 1 and figure 1.

Conclusion(s): The study quantifies the existing infrastructure and resource availability in Spain and Portugal's HTCs. Effective gene therapy implementation for PwH will necessitate planned investment by each country’s health service. Based on our findings, we recommend enhancing HTCs' expertise in gene therapy and adopting a "hub and spoke" model for efficient management of GT for PwH across both nations.