Wolfgang Miesbach: Roctavian Experience in Modern Hemophilia Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article by Flora Peyvandi et al., published in RPTH, adding:
“From a ‘haemophilia‑free mind’ to future‑ready care: why gene therapy treaters’ Roctavian experience still matters.
10 haemophilia gene therapy treaters from Germany, Italy and the United States share their real‑world experience with more than 25 adults with severe haemophilia A who received valoctocogene roxaparvovec (Roctavian):
- How they identify and prepare candidates (AAV5 testing, liver assessment, shared decision‑making)
- How centres and multidisciplinary teams are set up (including hub‑and‑spoke models, telemedicine and social work support)
- What actually happens on ‘infusion day’ and in the weeks afterwards
- How long‑term FVIII and liver monitoring is organised and how ALT rises are managed with steroids.
Why is this still important, even though Roctavian will no longer be available?
- The infrastructure they have built – hub‑and‑spoke networks, remote monitoring and close collaboration with hepatology and psychology – will underpin future programmes.
- Patients’ experiences (freedom from prophylaxis, fewer injections, greater independence, easier travel) and their concerns (FVIII durability, liver health, monitoring burden) offer valuable insights for future gene therapy discussions.
- Their collective message: many perceived barriers (approvals, logistics, training) proved manageable once clear pathways and communication structures were in place.”
Title: Real-world provider experiences with hemophilia A gene therapy: administration of valoctocogene roxaparvovec
Authors: Flora Peyvandi, Robert Klamroth, Johannes Oldenburg, Anita Rajasekhar, Andrea Buxton, Matteo Nicola Dario Di Minno, Saskia Gottstein, Amit Soni, Ezio Zanon, Andrew D. Leavitt
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