Objectives Adult onset Alexander Disease (AxD) is a rare leukodystrophy for which there is currently no treatment. Ceftriaxone has been proposed as a potential treatment for AxD. Here we report the clinical outcome of an extended course of intravenous cyclical ceftriaxone therapy in two patients with AxD.
Methods Case 1 is a 64 year old female with a five year history of progressive gait disturbance, bulbar palsy and horizontal diplopia. Case 2 is an 80 year old male with a two-year history of bulbar palsy and gait disturbance. Both cases were confirmed to have AxD and received intravenous ceftriaxone 2g daily for three weeks per month during the initial four months, then for 15 days monthly thereafter. Patients were assessed at baseline and approximately 4-month intervals using the Kurtzke Expanded Disability Status Scale, Modified Ranking Scale, and neurological examination.
Results Both cases displayed functional decline on ceftriaxone therapy, as assessed by outcome measures of disability. Progression on neuroimaging was also observed on MRI brain for both patients.
Conclusions These results suggest that ceftriaxone for AxD may not prevent functional decline. Caution should be applied before suggesting ceftriaxone for the management of AxD.
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