Razionale scientifico

The therapeutic landscape of hemophilia is undergoing a profound transformation. While the field is rapidly expanding beyond conventional replacement therapy toward rebalancing strategies, gene therapy, and gene editing, replacement treatment itself is also entering a new phase of innovation. In particular, the availability of novel factor replacement approaches, including high-sustained factor VIII therapies such as efanesoctocog alfa, is reshaping expectations in prophylaxis, bleed protection, treatment personalization, and quality of life. These advances are broadening the clinical possibilities within replacement therapy and are redefining how clinicians position factor-based strategies in relation to non-replacement options. At the same time, the emergence of rebalancing therapies, RNA interference approaches, AAV-based gene therapy, and gene editing is creating an increasingly complex decision-making environment for hemophilia treatment centers. Key issues now include treatment selection, patient profiling, monitoring, long-term safety, durability of response, access, sustainability, and future care pathways. Webinar Garden 2026 aims to provide a multidisciplinary update on the most recent advances, integrating clinical evidence, real-world data, international consensus recommendations, and the patient perspective, with the goal of outlining the future management of coagulation disorders in the era of advanced therapies.

Programma

Responsabili del corso

• Cedric Hermans

  Responsabile scientifico

  Specializzazione: Emostasi, trombosi, emofilia

  Struttura: Division of Haematology, Cliniques universitaires Saint-Luc, Catholic University of Louvain, Brussels, Belgium

  Ruolo: Full Professor, Head Haemostasis and Thrombosis Unit

• Aurelio Maggio

  Responsabile scientifico

  Specializzazione: Ematologia

  Struttura: Fondazione Franco e Piera Cutino ETS, Palermo

  Ruolo: Retired

• Mariasanta Napolitano

  Responsabile scientifico

  Specializzazione: Emofilia, malattie rare del sangue

  Struttura: Haematology and Rare Disease University Unit, Hospital V.Cervello, Palermo

  Ruolo: Head of Haematology and Rare Disease University Unit; Associate Professor of Hematology; Director specialization school in hematology, University of Palermo, PROMISE Department

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