Treatment-induced remission of medulloblastoma using a chemotherapeutic regimen devoid of vincristine in a child with Charcot-Marie-Tooth disease

Curr Oncol. 2019 Apr;26(2):e266-e269. doi: 10.3747/co.26.4491. Epub 2019 Apr 1.


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.

Keywords: Charcot–Marie–Tooth; chemotherapy; medulloblastoma; neurotoxicity; radiotherapy; vincristine.

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Carboplatin / therapeutic use
  • Cerebellar Neoplasms / therapy*
  • Charcot-Marie-Tooth Disease / drug therapy*
  • Chemoradiotherapy*
  • Child, Preschool
  • Cisplatin / therapeutic use
  • Cyclophosphamide / therapeutic use
  • Female
  • Humans
  • Lomustine / therapeutic use
  • Medulloblastoma / drug therapy*
  • Remission Induction


  • Antineoplastic Agents
  • Lomustine
  • Cyclophosphamide
  • Carboplatin
  • Cisplatin