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CSL EXPERT HIVE

Science updates

12 Jul 2024

WFH 2024 catch-up: latest insights on gene therapy and the patient journey in bleeding disorders

Pathology:

Haemophilia A Haemophilia B Von Willebrand Disease

In April, the World Federation of Hemophilia (WFH) Congress 2024 took place in Madrid, Spain, where CSL Behring sponsored the symposium 'Optimizing the patient journey in von Willebrand disease: Experiences and learnings as the navigator' and supported with the WFH-organised symposium 'Living the gene therapy treatment journey'.

Nicola Curry (UK), Heleen Eising (The Netherlands), Julia Rauscher (Austria) and Sunny Maini (UK) led the interactive symposium on von Willebrand disease (VWD), which focused on real-life experiences of stakeholders navigating diagnostic and care pathways. The multidisciplinary expert faculty shared their experiences travelling along the diagnostic pathway and invited the audience to identify issues in, and suggest improvements to, the patient journey in VWD. Findings from the interactive audience polls can be found here.

If you were unable to attend, or would like to watch the symposium again, it is available on-demand on the HAEMExpert Congress symposia tab.

In the WFH-organized symposium, available on-demand here, Cedric Hermans (Belgium), Greta Mulders (The Netherlands), Brian O'Mahoney (Ireland), Niclas Nilsson (Sweden) and Paul Thompson (UK) led an exciting discussion on the gene therapy treatment journey. Perspectives from both healthcare professionals and patients were shared, which stimulated an enriching and thought-provoking discussion.

In the Scientific Sessions, new data were presented from the HOPE-B clinical trial, which investigated etranacogene dezaparvovec gene therapy in patients with haemophilia B. The abstract 'Response to gene therapy in haemophilia B: Ranges of FIX sustained after 3 years in HOPE-B etranacogene dezaparavovec gene therapy' reported long-term data from HOPE-B, and demonstrated that stable factor IX expression is maintained for three years after a single infusion of etranacogene dezaparvovec. The abstract 'Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: Effects on patient-oriented outcomes measured using the Patient Repoted Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study' also reported three-year data from HOPE-B, but focused on the positive patient-reported impact of etranacogene dezaparvovec on quality of life.

Data from other CSL Behring-funded studies were also presented, on topics ranging from the haemophilia gene therapy patient journey to the value contribution of etranacogene dezaparvovec. If you would like to read any of the published abstracts from the WFH Congress, they are available here.