Journal
PEDIATRIC BLOOD & CANCER
Volume 69, Issue 8, Pages -Publisher
WILEY
DOI: 10.1002/pbc.29633
Keywords
neurofibromatosis type 1; paediatric; plexiform neurofibroma; selumetinib
Categories
Funding
- Canadian Cancer Society Research Institute
- Canadian Institutes of Health Research
- Sick Kids Foundation
- Garron Family Cancer Center
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This study reports a case series of 19 children treated with selumetinib, showing significant clinical benefit with symptom improvement or stabilization and acceptable toxicity profiles. This real-world experience confirms the previous trials' findings on the efficacy of selumetinib for this patient population.
Neurofibromatosis type 1-associated plexiform neurofibromas can cause debilitating symptoms and be life threatening. Treatment options are limited, given their tendency to regrow following surgery and their propensity to transform into malignant tumours following radiation. Selumetinib is an oral selective inhibitor of RAS-mitogen-activated protein kinase (MAPK) 1 and 2, which has shown efficacy for tumour shrinkage/stabilisation and symptom improvement. We report a national case series of 19 children treated with selumetinib. All patients experienced symptom improvement or stabilisation with an acceptable toxicity profile, including those patients previously treated with trametinib. This real-world experience confirms previous trials showing significant clinical benefit for this patient population.
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