1 CP Place (United States)

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Objective: To describe a patient with Leukoencephalopathy with cerebral calcifications and cysts (LCCC), and response to treatment with the vascular endothelial growth factor inhibitor (VEGF) bevacizumab. Background: LCCC is a childhood-onset disorder that causes diffuse white matter lesions, calcifications, and cysts to accumulate in the brain, leading to progressive neurological disability, including encephalopathy, dystonia, and quadriplegia. Although the cause of this disorder has yet to be identified, there is evidence that it is a genetic disease distinct from the clinically similar Coats-plus syndrome, and both disorders are thought to be microvasculopathies. Bevacizumab has been shown to reduce subretinal fluid and improve vision in a number of studies of patients with Coats disease, which is restricted to the eye, but its effects on cerebral microvasculopathies have not been studied. Design/Methods: We describe a patient with LCCC, who has been treated over the course of one year with monthly bevacizumab infusions. Informed consent was obtained from both the patient and his parents before treatment. We performed pre- and post-treatment neurological exams and brain magnetic resonance imaging (MRI) to assess response to bevacizumab. Results: The patient was born prematurely (29 weeks) and was globally developmentally delayed, walking at 21 months and talking at 5 years of age. He regressed from age seven, developing progressive spastic quadriplegia and dystonia, and MRI imaging showing accumulation of intracranial cysts, calcifications and white matter abnormalities through age 18. He was started on bevacizumab at age 18, and after 5 months, MRI showed decreased cyst size and decreased white matter signal abnormality. He showed clinical improvement during a year of treatment, without regression or complications related to bevacizumab. Conclusion: Our patient’s favorable response to VEGF inhibition indicates that this treatment may have a stabilizing effect on disease course, and a larger study is warranted.