The first year of treatment with selumetinib for NF1-related plexiform neurofibroma is medically challenging in real-life settings

Our efficacy results at 19.5 months for selumetinib show a genuine but modest reduction in PNF volume. The treatment has a good safety profile. While it is very common for adverse events to be reported at the start of treatment, they are of moderate severity and are managed by dose reduction or simple symptomatic treatment. Starting treatment early in life appears to lead to a more favorable outcome and should be considered.