Case study reveals successful high-risk treatment escalation in a child with ALL and Charcot-Marie-Tooth disease, suggesting alternatives to vincristine.
Vincristine is a cornerstone of acute lymphoblastic leukemia (ALL) therapy but poses a high risk of neurotoxicity in patients with hereditary neuropathies such as Charcot-Marie-Tooth disease (CMT). We report a pediatric patient with newly diagnosed ALL and incidentally discovered hereditary neuropathy with liability to pressure palsies (HNPP), a subtype of CMT. Vincristine was promptly discontinued, and treatment was successfully escalated to a high-risk protocol utilizing vincristine-sparing agents, including high-dose methotrexate. The patient achieved long-term remission without neurologic sequelae. This proactive, vincristine-sparing approach maintains full treatment intensity while minimizing risk of neurotoxicity and may be a practical option in similar cases.
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Thadani et al. (2025) studied this question.
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