TY  - INPR
TI  - MEK inhibition appears to improve symptom control in primary NRAS-driven CNS melanoma in children.
Y1  - 2017/03/02/
SN  - 1532-1827
AV  - public
N2  - 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.British Journal of Cancer advance online publication, 2 March 2017; doi:10.1038/bjc.2017.49 www.bjcancer.com.
ID  - discovery1543991
UR  - http://dx.doi.org/10.1038/bjc.2017.49
N1  - This work is licensed under the Creative Commons Attribution 4.0 International License.
To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
KW  - Melanoma; CNS; NRAS; trametinib; MEK inhibitor; paediatric; congenital melanocytic naevus
JF  - British Journal of Cancer
A1  - Kinsler, VA
A1  - O'Hare, P
A1  - Jacques, T
A1  - Hargrave, D
A1  - Slater, O
ER  -