Case report: Spontaneous improvement and treatment considerations in leukoencephalopathy with calcifications and cysts

被引:0
|
作者
Beerepoot, Shanice [1 ,2 ,5 ]
Ardon, Hilko [3 ,5 ]
Niers, Anneke [4 ,5 ]
van der Knaap, Marjo S. [1 ,2 ,5 ]
机构
[1] Univ Amsterdam, Emma Childrens Hosp, Amsterdam Leukodystrophy Ctr, Dept Child Neurol,Med Ctr, Amsterdam, Netherlands
[2] Univ Amsterdam, Amsterdam Neurosci Cellular & Mol Mech, Med Ctr, Amsterdam, Netherlands
[3] St Elizabeth Hosp, Dept Neurosurg, Tilburg, Netherlands
[4] Univ Amsterdam, Dept Neuro Oncol, Med Ctr, Amsterdam, Netherlands
[5] Vrije Univ, Ctr Neurogenom & Cognit Res, Dept Integrat Neurophysiol, Amsterdam, Netherlands
关键词
Bevacizumab; Leukoencephalopathy; Calcifications; Cysts; Spontaneous remission; DISORDER;
D O I
10.1016/j.clineuro.2024.108517
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
We present the case of a patient with leukoencephalopathy with calcifications and cysts (LCC), who experienced progressive severe hemiparesis despite multiple neurosurgical interventions of a large contralateral cyst. Bevacizumab was proposed as an ultimate treatment option based on prior case reports. While awaiting reimbursement approval for bevacizumab, major improvement occurred in both clinical and radiological disease manifestations. The disease course of LCC is variable and unpredictable; neurosurgical treatment should be reserved for severe and progressive neurological deficits. Bevacizumab has been reported as a promising alternative treatment option. Importantly, in our case the observed clinical improvement would have been attributed to the effects of bevacizumab, if started when requested. Our case underscores the need for a natural history study for LCC and the necessity of validating treatment efficacy by systematic evaluation through appropriate clinical trials rather than relying on anecdotal evidence from published case reports.
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页数:4
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