Treatment-induced remission of medulloblastoma using a chemotherapeutic regimen devoid of vincristine in a child with charcot–marie–tooth disease

Academic Article


  • Charcot–Marie–Tooth (cmt) disease is the most common form of inherited neuropathy. Core features include peripheral neuropathy and secondary axonal degeneration, with a noted distal predominance of limb-muscle wasting, weakness, and sensory loss. Given the significant prevalence of cmt, superimposed neoplastic disease can be encountered within this patient population. Malignancies that are treated with vincristine (a microtubule-targeting agent), even at low doses as part of standard treatment, pose a significant challenge for patients with cmt. Here, we present the case of a child with cmt who was successfully treated for medulloblastoma without vincristine, a standard drug used for treatment of that disease, to avoid the risk of severe debilitating neuropathy. This report is the first of a patient successfully treated for medulloblastoma without vincristine.
  • Digital Object Identifier (doi)

    Author List

  • Bernstock JD; Cohen JL; Singh S; Schlappi CW; Fiveash JB; Johnston JM; Fequiere P; Orr BA; Li R; Friedman GK
  • Start Page

  • e266
  • End Page

  • e269
  • Volume

  • 26
  • Issue

  • 2