eISSN: 1509-572x
ISSN: 1641-4640
Folia Neuropathologica
Current issue Archive Manuscripts accepted About the journal Special Issues Editorial board Reviewers Abstracting and indexing Subscription Contact Instructions for authors Ethical standards and procedures
Editorial System
Submit your Manuscript
SCImago Journal & Country Rank
vol. 61
Case report

Off-label use of eculizumab for neurological symptoms and progressive vision loss

Alicja Kuźniewska
Marta Jaskólska
Mariusz Kwarciany
Aleksandra Ciarka
Rafał Pęksa
Zbigniew Zdrojewski
Marcin Okrój

Department of Cell Biology and Immunology, Intercollegiate Faculty of Biotechnology, University of Gdańsk and Medical University of Gdańsk, Poland
Department of Rheumatology, Clinical Immunology, Geriatrics and Internal Medicine, Medical University of Gdańsk, Poland
Department of Neurology, Division of Adult Neurology, Medical University of Gdańsk, Poland
Department of Pathomorphology, Medical University of Gdańsk, Poland
Folia Neuropathol 2023; 61 (4):442-447
Online publish date: 2023/12/29
Article file
- Off-label.pdf  [0.27 MB]
Get citation
PlumX metrics:
1. Annane D, Heming N, Grimaldi-Bensouda L, Frémeaux-Bacchi V, Vigan M, Roux AL, Marchal A, Michelon H, Rottman M, Moine P; Garches COVID 19 Collaborative Group. Eculizumab as an emergency treatment for adult patients with severe COVID-19 in the intensive care unit: A proof-of-concept study. EClinicalMedicine 2020; 28: 100590.
2. Blom AM, Corvillo F, Magda M, Stasiłojć G, Nozal P, PérezValdivia MÁ, Cabello-Chaves V, Rodríguez de Córdoba S, López-Trascasa M, Okrój M. Testing the activity of complement convertases in serum/plasma for diagnosis of C4NeF-mediated C3 glomerulonephritis. J Clin Immunol 2016; 36: 517-527.
3. Blom AM, Österborg A, Mollnes TE, Okroj M. Antibodies reactive to cleaved sites in complement proteins enable highly specific measurement of soluble markers of complement activation. Mol Immunol 2015; 66: 164-170.
4. Edwards AO, Ritter R, Abel KJ, Manning A, Panhuysen C, Farrer LA. Complement factor H polymorphism and age-related macular degeneration. Science 2005; 308: 421-424.
5. Garred P, Tenner AJ, Mollnes TE. Therapeutic targeting of the complement system: From rare diseases to pandemics. Pharmacol Rev 2021; 73: 792-827.
6. Gastoldi S, Aiello S, Galbusera M, Breno M, Alberti M, Bresin E, Mele C, Piras R, Liguori L, Santarsiero D, Benigni A, Remuzzi G, Noris M. An ex vivo test to investigate genetic factors conferring susceptibility to atypical haemolytic uremic syndrome. Front Immunol 2023; 14: 1112257.
7. Hebecker M, Jozsi M. Factor H-related protein 4 activates complement by serving as a platform for the assembly of alternative pathway C3 convertase via its interaction with C3b protein. J Biol Chem 2012; 287: 19528-19536.
8. Herbert AP, Deakin JA, Schmidt CQ, Blaum BS, Egan C, Ferreira VP, Pangburn MK, Lyon M, Uhrín D, Barlow PN. Structure shows that a glycosaminoglycan and protein recognition site in factor H is perturbed by age-related macular degeneration-linked single nucleotide polymorphism. J Biol Chem 2007; 282: 18960-18968.
9. Java A, Baciu P, Widjajahakim R, Sung YJ, Yang J, Kavanagh D, Atkinson J, Seddon J. Functional analysis of rare genetic variants in complement factor I (CFI) using a serum-based assay in advanced age-related macular degeneration. Transl Vis Sci Technol 2020; 9: 37.
10. Kim MS, Prasad V. The clinical trials portfolio for on-label and off-label studies of eculizumab. JAMA Intern Med 2020; 180: 315-317.
11. Kurolap A, Eshach Adiv O, Hershkovitz T, Tabib A, Karbian N, Paperna T, Mory A, Vachyan A, Slijper N, Steinberg R, Zohar Y, Mevorach D, Baris Feldman H. Eculizumab is safe and effective as a long-term treatment for protein-losing enteropathy due to CD55 deficiency. J Pediatr Gastroenterol Nutr 2019; 68: 325-333.
12. Merle NS, Church SE, Fremeaux-Bacchi V, Roumenina LT. Complement system part I – molecular mechanisms of activation and regulation. Front Immunol 2015; 6: 262.
13. Norville K, Stephen J, Mead-Harvey C, Corey R, Votruba C. Evaluation of eculizumab use in renal transplant recipients. Prog Transplant 2023; 33: 162-167.
14. Oskam N, Damelang T, Streutker M, Ooijevaar-de Heer P, Nouta J, Koeleman C, Van Coillie J, Wuhrer M, Vidarsson G, Rispens T. Factors affecting IgG4-mediated complement activation. Front Immunol 2023; 14: 1087532.
15. Rodríguez de Córdoba S. Genetic variability shapes the alternative pathway complement activity and predisposition to complement-related diseases. Immunol Rev 2023; 313: 71-90.
16. Roy B, Nowak RJ. Eculizumab in acute motor axonal neuropathy: An ethical application of an off-label indication. J Clin Neuromuscul Dis 2020; 22: 63-64.
Copyright: © 2023 Mossakowski Medical Research Centre Polish Academy of Sciences and the Polish Association of Neuropathologists. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International (CC BY-NC-SA 4.0) License (http://creativecommons.org/licenses/by-nc-sa/4.0/), allowing third parties to copy and redistribute the material in any medium or format and to remix, transform, and build upon the material, provided the original work is properly cited and states its license.
Quick links
© 2024 Termedia Sp. z o.o.
Developed by Bentus.