Chapter title |
Ceftriaxone for Alexander’s Disease: A Four-Year Follow-Up
|
---|---|
Chapter number | 180 |
Book title |
JIMD Reports – Case and Research Reports, 2012/6
|
Published in |
JIMD Reports, October 2012
|
DOI | 10.1007/8904_2012_180 |
Pubmed ID | |
Book ISBNs |
978-3-64-235517-2, 978-3-64-235518-9
|
Authors |
GianPietro Sechi, Isabella Ceccherini, Tiziana Bachetti, Giovanni A. Deiana, Elia Sechi, Pietro Balbi, Sechi, GianPietro, Ceccherini, Isabella, Bachetti, Tiziana, Deiana, Giovanni A., Sechi, Elia, Balbi, Pietro |
Abstract |
In 2010, we reported the successful clinical outcome related to a 20-month course of intravenous, cyclical ceftriaxone, in a patient with adult-onset Alexander's disease. We now provide evidence that the progression of the patient's signs/symptoms was halted and reversed with a 4-year-long extension of the trial.The patient's clinical signs/symptoms were evaluated before the start and every 6 months for 6 years. For the early 2 years, without therapy, and for the following 4 years, after intravenous ceftriaxone 2 g daily, for 3 weeks monthly during the initial 4 months, then for 15 days monthly.Gait ataxia and dysarthria were assessed clinically on a 0 to 4 scale. Palatal myoclonus and nystagmus/oscillopsia were monitored by videotape and a self-evaluation scale. The degree of disability, measured by a modified Rankin scale, and the brain MRI were periodically evaluated.Before ceftriaxone therapy, in a 2-year period, gait ataxia and dysarthria worsened from mild to marked, palatal myoclonus spread from the soft palate to lower facial muscles, and the patient complained of oscillopsia. After 4 years of ceftriaxone therapy, gait ataxia and dysarthria improved, from marked to mild at clinical rating scales. The palatal myoclonus was undetectable; the patient did not complained of oscillopsia and declared a progressively better quality of life. Ceftriaxone was safe.This case report provides Class IV evidence that intravenous cycles of ceftriaxone may halt and/or reverse the progression of neurodegeneration in patients with adult-onset Alexander's disease and may significantly improve their quality of life. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 24 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 5 | 21% |
Researcher | 4 | 17% |
Student > Ph. D. Student | 3 | 13% |
Student > Doctoral Student | 2 | 8% |
Other | 2 | 8% |
Other | 2 | 8% |
Unknown | 6 | 25% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 4 | 17% |
Biochemistry, Genetics and Molecular Biology | 3 | 13% |
Nursing and Health Professions | 3 | 13% |
Neuroscience | 2 | 8% |
Arts and Humanities | 1 | 4% |
Other | 1 | 4% |
Unknown | 10 | 42% |