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Uneventful administration of vincristine in Charcot–Marie–Tooth disease type 1X

Charcot–Marie–Tooth (CMT) disease is the most common inherited peripheral neuropathy. Administration of vinca alkaloids is considered contraindicated in CMT because of the risk of life threatening acute neuropathy. We describe a 5‐year‐old patient with a family history of X‐linked CMT1 who presented with Wilms tumour. Despite confirmation of a connexin 32 point mutation, the patient was treated with 40.5 mg/m2 of vincristine with no clinical evidence of drug‐related neuropathy. This case describes vincristine being safely administered to a patient with molecularly proven type CMT 1X, but in type 1A CMT vincristine is still contraindicated. An extensive literature review revealed no other series of cases in which vincristine administration in molecularly proven CMT 1X had been described. CMT should be excluded in any patient who develops a profound, acute neuropathy following vincristine, as many patients in the cases reviewed were asymptomatic prior to treatment. Pediatr Blood Cancer 2008;50:874–876. © 2007 Wiley‐Liss, Inc.