Treatment-Induced Remission of Medulloblastoma Using a Chemotherapeutic Regimen Devoid of Vincristine in a Child with Charcot–Marie–Tooth Disease

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.