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Br J Cancer. 2017 Apr 11;116(8):990-993. doi: 10.1038/bjc.2017.49. Epub 2017 Mar 2.

MEK inhibition appears to improve symptom control in primary NRAS-driven CNS melanoma in children.

Author information

1
Paediatric Dermatology, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, UK.
2
Genetics and Genomic Medicine, UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.
3
Paediatric Oncology, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, UK.
4
Paediatric Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, UK.
5
Developmental Biology and Cancer Programme, UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.

Abstract

BACKGROUND:

Primary melanoma of the CNS in children is extremely rare, and usually linked to congenital melanocytic naevus syndrome, caused by mosaicism for oncogenic NRAS mutations. Outcome is fatal in all cases. Data from murine and in vitro studies suggest that MEK inhibition is a possible therapeutic option.

METHODS:

Four children with NRAS-mutated CNS melanoma were treated with Trametinib on a compassionate basis.

RESULTS:

All four had an improvement in symptoms and objectively in signs. These varied from mild improvement for 1 month, to a sustained symptom-free period of 9 months in one case. In all cases there was eventual disease progression through treatment, followed by rapid death after discontinuation. There were no clinically-significant side effects.

CONCLUSIONS:

Trametinib is the first therapy to show any objective or measurable effect in NRAS-mutated primary CNS melanoma, with few side effects in this small series. The role of this therapy should be explored further in this rare paediatric tumour.

PMID:
28253523
PMCID:
PMC5396107
DOI:
10.1038/bjc.2017.49
[Indexed for MEDLINE]
Free PMC Article

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