Eculizumab use in neuromyelitis optica spectrum disorders: routine clinical care data from a european cohort
Background and Objectives - Attack prevention is crucial in managing neuromyelitis optica spectrum disorders (NMOSDs). Eculizumab (ECU), an inhibitor of the terminal complement cascade, was highly effective in preventing attacks in a phase III trial of aquaporin-4 (AQP4)-IgG seropositive(+) NMOSDs....
Gespeichert in:
| Hauptverfasser: | , , , , |
|---|---|
| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
October 1, 2024
|
| In: |
Neurology
Year: 2024, Jahrgang: 103, Heft: 9, Pages: 1-16 |
| ISSN: | 1526-632X |
| DOI: | 10.1212/wnl.0000000000209888 |
| Online-Zugang: | Verlag, kostenfrei, Volltext: https://doi.org/10.1212/wnl.0000000000209888 Resolving-System, kostenfrei: https://doi.org/10.25673/117826 Verlag, kostenfrei, Volltext: https://www.neurology.org/doi/10.1212/WNL.0000000000209888 
|
| Verfasserangaben: | Marius Ringelstein, Alexander Emmer, Sven Jarius, Mirjam Korporal-Kuhnke, Brigitte Wildemann [und viele weitere] |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1913946495 | ||
| 003 | DE-627 | ||
| 005 | 20250624191918.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 250109s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1212/wnl.0000000000209888 |2 doi | |
| 024 | 7 | |a 10.25673/117826 |2 doi | |
| 035 | |a (DE-627)1913946495 | ||
| 035 | |a (DE-599)KXP1913946495 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 33 |2 sdnb | ||
| 100 | 1 | |a Ringelstein, Marius |d 1977- |e VerfasserIn |0 (DE-588)136773559 |0 (DE-627)694645745 |0 (DE-576)301269084 |4 aut | |
| 245 | 1 | 0 | |a Eculizumab use in neuromyelitis optica spectrum disorders |b routine clinical care data from a european cohort |c Marius Ringelstein, Alexander Emmer, Sven Jarius, Mirjam Korporal-Kuhnke, Brigitte Wildemann [und viele weitere] |
| 264 | 1 | |c October 1, 2024 | |
| 300 | |b Diagramme | ||
| 300 | |a 6 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Gesehen am 24.06.2025 | ||
| 520 | |a Background and Objectives - Attack prevention is crucial in managing neuromyelitis optica spectrum disorders (NMOSDs). Eculizumab (ECU), an inhibitor of the terminal complement cascade, was highly effective in preventing attacks in a phase III trial of aquaporin-4 (AQP4)-IgG seropositive(+) NMOSDs. In this article, we evaluated effectiveness and safety of ECU in routine clinical care. - Methods - We retrospectively evaluated patients with AQP4-IgG+ NMOSD treated with ECU between December 2014 and April 2022 at 20 German and 1 Austrian university center(s) of the Neuromyelitis Optica Study Group (NEMOS) by chart review. Primary outcomes were effectiveness (assessed using annualized attack rate [AAR], MRI activity, and disability changes [Expanded Disability Status Scale {EDSS}]) and safety (including adverse events, mortality, and attacks after meningococcal vaccinations), analyzed by descriptive statistics. - Results - Fifty-two patients (87% female, age 55.0 ± 16.3 years) received ECU for 16.2 (interquartile range [IQR] 9.6 - 21.7) months. Forty-five patients (87%) received meningococcal vaccination before starting ECU, 9 with concomitant oral prednisone and 36 without. Seven of the latter (19%) experienced attacks shortly after vaccination (median: 9 days, IQR 6-10 days). No postvaccinal attack occurred in the 9 patients vaccinated while on oral prednisone before starting ECU and in 25 (re-)vaccinated while on ECU. During ECU therapy, 88% of patients were attack-free. The median AAR decreased from 1.0 (range 0-4) in the 2 years preceding ECU to 0 (range 0-0.8; p < 0.001). The EDSS score from start to the last follow-up was stable (median 6.0), and the proportion of patients with new T2-enhancing or gadolinium-enhancing MRI lesions in the brain and spinal cord decreased. Seven patients (13%) experienced serious infections. Five patients (10%; median age 53.7 years) died on ECU treatment (1 from myocardial infarction, 1 from ileus with secondary sepsis, and 3 from systemic infection, including 1 meningococcal sepsis), 4 were older than 60 years and severely disabled at ECU treatment start (EDSS score ≥ 7). The overall discontinuation rate was 19%. - Discussion - Eculizumab proved to be effective in preventing NMOSD attacks. An increased risk of attacks after meningococcal vaccination before ECU start and potentially fatal systemic infections during ECU - particularly in patients with comorbidities - must be considered. Further research is necessary to explore optimal timing for meningococcal vaccinations. - Classification of Evidence - This study provides Class IV evidence that eculizumab reduces annualized attack rates and new MRI lesions in AQP4-IgG+ patients with NMOSD. | ||
| 540 | |q DE-3 |a Namensnennung - Nicht kommerziell - Keine Bearbeitungen 4.0 International |f CC BY-NC-ND 4.0 |2 cc |u https://creativecommons.org/licenses/by-nc-nd/4.0/ | ||
| 700 | 1 | |a Emmer, Alexander |d 1978- |e VerfasserIn |0 (DE-588)140326596 |0 (DE-627)703639633 |0 (DE-576)316432423 |4 aut | |
| 700 | 1 | |a Jarius, Sven |e VerfasserIn |0 (DE-588)1054615918 |0 (DE-627)791654818 |0 (DE-576)410367478 |4 aut | |
| 700 | 1 | |a Korporal-Kuhnke, Mirjam |d 1975- |e VerfasserIn |0 (DE-588)129891851 |0 (DE-627)483343579 |0 (DE-576)297889583 |4 aut | |
| 700 | 1 | |a Wildemann, Brigitte |e VerfasserIn |0 (DE-588)110203844 |0 (DE-627)510150004 |0 (DE-576)171831330 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Neurology |d Philadelphia, Pa. : Wolters Kluwer, 1951 |g 103(2024), 9, Artikel-ID e209888, Seite 1-16 |h Online-Ressource |w (DE-627)302718524 |w (DE-600)1491874-2 |w (DE-576)079876609 |x 1526-632X |7 nnas |a Eculizumab use in neuromyelitis optica spectrum disorders routine clinical care data from a european cohort |
| 773 | 1 | 8 | |g volume:103 |g year:2024 |g number:9 |g elocationid:e209888 |g pages:1-16 |g extent:6 |a Eculizumab use in neuromyelitis optica spectrum disorders routine clinical care data from a european cohort |
| 856 | 4 | 0 | |u https://doi.org/10.1212/wnl.0000000000209888 |x Verlag |x Resolving-System |z kostenfrei |3 Volltext |
| 856 | 4 | 0 | |u https://doi.org/10.25673/117826 |x Resolving-System |z kostenfrei |
| 856 | 4 | 0 | |u https://www.neurology.org/doi/10.1212/WNL.0000000000209888 |x Verlag |z kostenfrei |3 Volltext |
| 951 | |a AR | ||
| 992 | |a 20250624 | ||
| 993 | |a Article | ||
| 994 | |a 2024 | ||
| 998 | |g 110203844 |a Wildemann, Brigitte |m 110203844:Wildemann, Brigitte |d 910000 |d 911100 |d 50000 |e 910000PW110203844 |e 911100PW110203844 |e 50000PW110203844 |k 0/910000/ |k 1/910000/911100/ |k 0/50000/ |p 26 | ||
| 998 | |g 129891851 |a Korporal-Kuhnke, Mirjam |m 129891851:Korporal-Kuhnke, Mirjam |d 910000 |d 911100 |e 910000PK129891851 |e 911100PK129891851 |k 0/910000/ |k 1/910000/911100/ |p 25 | ||
| 998 | |g 1054615918 |a Jarius, Sven |m 1054615918:Jarius, Sven |d 910000 |d 911100 |e 910000PJ1054615918 |e 911100PJ1054615918 |k 0/910000/ |k 1/910000/911100/ |p 24 | ||
| 999 | |a KXP-PPN1913946495 |e 4738298070 | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"origin":[{"dateIssuedDisp":"October 1, 2024","dateIssuedKey":"2024"}],"relHost":[{"origin":[{"publisher":"Wolters Kluwer ; Ovid ; Lippincott Williams & Wilkins","dateIssuedKey":"1951","dateIssuedDisp":"1951-","publisherPlace":"Philadelphia, Pa. ; [Erscheinungsort nicht ermittelbar] ; Hagerstown, Md."}],"pubHistory":["1.1951 -"],"language":["eng"],"type":{"bibl":"periodical","media":"Online-Ressource"},"note":["Gesehen am 12.12.23"],"id":{"eki":["302718524"],"issn":["1526-632X"],"zdb":["1491874-2"]},"title":[{"subtitle":"official journal of the American Academy of Neurology","title_sort":"Neurology","title":"Neurology"}],"part":{"pages":"1-16","extent":"6","text":"103(2024), 9, Artikel-ID e209888, Seite 1-16","issue":"9","year":"2024","volume":"103"},"physDesc":[{"extent":"Online-Ressource"}],"recId":"302718524","disp":"Eculizumab use in neuromyelitis optica spectrum disorders routine clinical care data from a european cohortNeurology"}],"person":[{"display":"Ringelstein, Marius","family":"Ringelstein","given":"Marius","role":"aut"},{"display":"Emmer, Alexander","family":"Emmer","given":"Alexander","role":"aut"},{"display":"Jarius, Sven","given":"Sven","role":"aut","family":"Jarius"},{"display":"Korporal-Kuhnke, Mirjam","family":"Korporal-Kuhnke","role":"aut","given":"Mirjam"},{"family":"Wildemann","given":"Brigitte","role":"aut","display":"Wildemann, Brigitte"}],"language":["eng"],"type":{"bibl":"article-journal","media":"Online-Ressource"},"note":["Gesehen am 24.06.2025"],"title":[{"title":"Eculizumab use in neuromyelitis optica spectrum disorders","title_sort":"Eculizumab use in neuromyelitis optica spectrum disorders","subtitle":"routine clinical care data from a european cohort"}],"physDesc":[{"noteIll":"Diagramme","extent":"6 S."}],"recId":"1913946495","name":{"displayForm":["Marius Ringelstein, Alexander Emmer, Sven Jarius, Mirjam Korporal-Kuhnke, Brigitte Wildemann [und viele weitere]"]},"id":{"doi":["10.1212/wnl.0000000000209888","10.25673/117826"],"eki":["1913946495"]}} | ||
| SRT | |a RINGELSTEIECULIZUMAB1202 | ||