Trametinib therapy for children with neurofibromatosis type 1 and life-threatening plexiform neurofibroma or treatment-refractory low-grade glioma.
Rebecca RonsleyCeline D HounjetSylvia ChengShahrad Rod RassekhWalter J DuncanChristopher DunhamJane GardinerArvindera GhagJeffrey P LudemannDavid WensleyWingfield RehmusMichael A SargentJuliette HukinPublished in: Cancer medicine (2021)
Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF-1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.
Keyphrases
- low grade
- clinical trial
- young adults
- end stage renal disease
- high grade
- ejection fraction
- chronic kidney disease
- prognostic factors
- peritoneal dialysis
- randomized controlled trial
- machine learning
- combination therapy
- patient reported outcomes
- big data
- immune response
- artificial intelligence
- patient reported
- placebo controlled