Off-label use of eculizumab for neurological symptoms and progressive vision loss
We describe the results of eculizumab treatment of a patient with pachymeningitis, inflammatory infiltration of the left frontal lobe, and cerebral hematoma, who presented with progressive vision loss, epileptic seizures, and abnormal pattern of the complement system parameters. A 30-year-old female...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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Termedia Publishing House
2023-12-01
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| Series: | Folia Neuropathologica |
| Subjects: | |
| Online Access: | https://www.termedia.pl/Off-label-use-of-eculizumab-for-neurological-symptoms-and-progressive-vision-loss,20,52237,1,1.html |
| _version_ | 1797333357735968768 |
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| author | Alicja Kuźniewska Marta Jaskólska Mariusz Kwarciany Aleksandra Ciarka Rafał Pęksa Zbigniew Zdrojewski Marcin Okrój |
| author_facet | Alicja Kuźniewska Marta Jaskólska Mariusz Kwarciany Aleksandra Ciarka Rafał Pęksa Zbigniew Zdrojewski Marcin Okrój |
| author_sort | Alicja Kuźniewska |
| collection | DOAJ |
| description | We describe the results of eculizumab treatment of a patient with pachymeningitis, inflammatory infiltration of the left frontal lobe, and cerebral hematoma, who presented with progressive vision loss, epileptic seizures, and abnormal pattern of the complement system parameters. A 30-year-old female patient, initially diagnosed with hypereosinophilia and a tumour of the left orbit, developed a significant visual impairment in the left eye, progressive vision loss in the right eye, and neurological symptoms in the form of epileptic seizures and behavioural changes. Magnetic resonance imaging (MRI) revealed thickening of the dura mater in the left frontal area, slight oedema of the cortex, and subcortical white matter. Orbit biopsy showed non-specific inflammatory infiltrates. Despite the initial good response, symptoms progressed during treatment with glucocorticoids and immunosuppressants. Increased activity of the alternative complement pathway accompanied by a low level of its main inhibitor, factor H (FH), and the presence of anti-FH autoantibodies, was found. Genetic analysis revealed several missense variants of complement proteins, including two disease-linked mutations in FH (p.H402Y) and FI (T300A). An attempt to apply a complement C5 blocker, eculizumab, has been made. Neurological symptoms subsided, vision loss was inhibited, laboratory parameters improved, and discontinuation of steroid therapy was possible. The case underlines the role of complement system dysregulation in neurological distress. |
| first_indexed | 2024-03-08T08:02:37Z |
| format | Article |
| id | doaj.art-73696faf1fa94dabad4b87299c8dff95 |
| institution | Directory Open Access Journal |
| issn | 1641-4640 1509-572X |
| language | English |
| last_indexed | 2024-03-08T08:02:37Z |
| publishDate | 2023-12-01 |
| publisher | Termedia Publishing House |
| record_format | Article |
| series | Folia Neuropathologica |
| spelling | doaj.art-73696faf1fa94dabad4b87299c8dff952024-02-02T11:53:59ZengTermedia Publishing HouseFolia Neuropathologica1641-46401509-572X2023-12-0161444244710.5114/fn.2023.13431352237Off-label use of eculizumab for neurological symptoms and progressive vision lossAlicja KuźniewskaMarta JaskólskaMariusz KwarcianyAleksandra CiarkaRafał PęksaZbigniew ZdrojewskiMarcin OkrójWe describe the results of eculizumab treatment of a patient with pachymeningitis, inflammatory infiltration of the left frontal lobe, and cerebral hematoma, who presented with progressive vision loss, epileptic seizures, and abnormal pattern of the complement system parameters. A 30-year-old female patient, initially diagnosed with hypereosinophilia and a tumour of the left orbit, developed a significant visual impairment in the left eye, progressive vision loss in the right eye, and neurological symptoms in the form of epileptic seizures and behavioural changes. Magnetic resonance imaging (MRI) revealed thickening of the dura mater in the left frontal area, slight oedema of the cortex, and subcortical white matter. Orbit biopsy showed non-specific inflammatory infiltrates. Despite the initial good response, symptoms progressed during treatment with glucocorticoids and immunosuppressants. Increased activity of the alternative complement pathway accompanied by a low level of its main inhibitor, factor H (FH), and the presence of anti-FH autoantibodies, was found. Genetic analysis revealed several missense variants of complement proteins, including two disease-linked mutations in FH (p.H402Y) and FI (T300A). An attempt to apply a complement C5 blocker, eculizumab, has been made. Neurological symptoms subsided, vision loss was inhibited, laboratory parameters improved, and discontinuation of steroid therapy was possible. The case underlines the role of complement system dysregulation in neurological distress.https://www.termedia.pl/Off-label-use-of-eculizumab-for-neurological-symptoms-and-progressive-vision-loss,20,52237,1,1.htmleculizumab complement system epilepsy cerebral hematoma vision loss |
| spellingShingle | Alicja Kuźniewska Marta Jaskólska Mariusz Kwarciany Aleksandra Ciarka Rafał Pęksa Zbigniew Zdrojewski Marcin Okrój Off-label use of eculizumab for neurological symptoms and progressive vision loss Folia Neuropathologica eculizumab complement system epilepsy cerebral hematoma vision loss |
| title | Off-label use of eculizumab for neurological symptoms and progressive vision loss |
| title_full | Off-label use of eculizumab for neurological symptoms and progressive vision loss |
| title_fullStr | Off-label use of eculizumab for neurological symptoms and progressive vision loss |
| title_full_unstemmed | Off-label use of eculizumab for neurological symptoms and progressive vision loss |
| title_short | Off-label use of eculizumab for neurological symptoms and progressive vision loss |
| title_sort | off label use of eculizumab for neurological symptoms and progressive vision loss |
| topic | eculizumab complement system epilepsy cerebral hematoma vision loss |
| url | https://www.termedia.pl/Off-label-use-of-eculizumab-for-neurological-symptoms-and-progressive-vision-loss,20,52237,1,1.html |
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