Drugs in context
Standalone NF1: guiding the transition from paediatric to adult care
By Salvador H, Lassaletta Á, López-Gutiérrez JC and 2 others.
Abstract
This report summarizes expert and patient-representative perspectives from the meeting entitled 'Standalone NF1: guiding the transition from paediatric to adult care', held in Madrid on October 2, 2025. Its primary objective was to examine clinical, organizational and patient-related challenges during transition and identify practical measures to improve continuity of care. Discussions focused on multidisciplinary co-ordination, case management, patient autonomy and structured transition pathways. NF1 characteristics, plexiform neurofibroma burden, surgery, and the currently approved MEK inhibitors selumetinib and mirdametinib, are summarized only for clinical context; selumetinib is discussed in greater detail because it was the treatment available at that time and the one addressed during the meeting.
Keywords
- MEK inhibitors
- malignant peripheral nerve sheath tumour
- multidisciplinary
- neurofibromatosis 1
- plexiform neurofibroma
- selumetinib
- transitional care
Source
Published in Drugs in context. The abstract is shown as published and remains the work of its authors; read the full article at the source.
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