Toxicology
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We have documented visual and auditory neurotoxicity in 42 of 89 patients with transfusion-dependent anemia who were receiving iron chelation therapy with subcutaneous deferoxamine (DFO). Of the affected groups, 13 presented with visual loss or deafness or both, and ophthalmologic, audiologic, and visual evoked potential studies (VEPs) uncovered abnormalities in 29 more. Four patients with visual loss had optic neuropathy with a marked decrease in acuity and loss of color vision. ⋯ Significantly lower doses of DFO were being taken by patients without abnormalities than by those with visual symptoms, abnormal audiograms, or prolonged VEPs (P less than 0.001, less than 0.006, and less than 0.04, respectively). The data implicate high-dose DFO as a central factor in the pathogenesis of the neurotoxicity. Our serial studies provide the basis for effective yet safe DFO administration for patients who require the agent.