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

J. D. Bernstock, J. L. Cohen, S. Singh, C. W. Schlappi, J. B. Fiveash, J. M. Johnston, P. Fequiere, B. A. Orr, R. Li, G. K. Friedman


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.


Charcot–Marie–Tooth, chemotherapy; medulloblastoma; neurotoxicity; radiotherapy; vincristine

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ISSN: 1198-0052 (Print) ISSN: 1718-7729 (Online)