Quick and sustained clinical response to MEK inhibitor I in a NF1 patient with neurofibromas

Author: Ecancermedicalscience. 2018; 12: 862.

Published On: 08/28/2018

Ecancer medical science reported their August 2018 issue about a case of a 12 year old child with NF1.  Some of the symptoms that were discussed is a large subcutaneous neurofibroma on the left hand leading to difficulty in using his hand and a gradual degradation of walking with scoliosis which eventually made it impossible to walk and invalidating pain appeared.  The child was treated with a MEK inhibitor (trametinib) for a phase I trial for one year.  Two months off trial dramatic clinical improvement with walking recovery, and disappearance of pain remain.  Softening of the mass in the left hand increased functionality.  Treatment was well tolerated. 

The article states,  “We do not believe that MEK inhibitor treatment will provide a substitute for standard antalgic treatment for patients with NF1, given the potential toxicity, the cost and the potential duration of treatment.  When other symptoms, such as those seen in the patient reported here, are presented and cannot be improved by other treatments, then the use of MEK inhibitors may represent a valuable alternative”.  Read the full article here.