Manuel Rodríguez-López, Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, Vigo, Pontevedra, España
M.ª Teresa Álvarez-Román, Servicio de Hematología y Hemoterapia, Hospital Universitario La Paz, IdiPaz, Universidad Autónoma, Madrid, España
Michael Calviño-Suárez, Servicio de Hematología y Hemoterapia, Complejo Hospitalario Universitario A Coruña, A Coruña, España
Carmen Albo-López, Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, EOXI Vigo, Vigo, Pontevedra; Instituto de Investigación Sanitaria Galicia-Sur, Vigo, Pontevedra; Esapaña
Ramiro J. Núñez-Vázquez, Unidad de Trombosis y Hemostasia, Hospital Universitario Virgen del Rocío, Sevilla, España
Raquel Ocampo-Martínez, Servicio de Hematología y Hemoterapia, Hospital Universitario Álvaro Cunqueiro, EOXI Vigo, Vigo, España
Carlos Pérez-Miguez, Grupo de Hematologia Computacional y Genomica del Instituto de Investigacion Sanitaria, Santiago de Compostela, España
Adrián Mosquera-Orgueira, Grupo de Hematologia Computacional y Genomica del Instituto de Investigacion Sanitaria, Santiago de Compostela; Servicio de Hematología y Hemoterapia, Complejo Hospitalario Universitario de Santiago de Compostela, Santiago de Compostela. España
Introduction: Gene therapy revolutionises treatment in haemophilia A, with promising results, but different safety and efficacy profiles. Objective: To indirectly compare efficacy and safety of valoctocogene roxaparvovec (VR) and giroctocogene fitelparvovec (GF). Material and method: Published data of VR and GF were analysed. Statistical tests proposed by ChatGPT-4o were used for comparisons. Results: FVIII levels > 5%, no significant differences at 24 months (p = 0.516). For total annualized bleeding rate, similar reductions without statistical significance (p = 0.263). GF showed less need for corticosteroids (p = 0.0012). 81.3% of RV patients had no bleeding, significantly higher than 64% of GF (p = 0.014). Conclusions: Both therapies are effective, with limitations. GF stands out for lower corticosteroid use and FVIII stability. RV has better control of spontaneous bleeding. These differences reflect challenges in clinical study design and comparison, and further evaluation is needed.
Keywords: Gene therapy. Hemophilia A. Valoctocogene. Roxaparvovec. Giroctocogene. Fitelparvovec.