Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab : subgroup analysis of REGAIN and its extension study
© 2021 Wiley Periodicals LLC..
INTRODUCTION/AIMS: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+ ) gMG previously treated with rituximab.
METHODS: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE.
RESULTS: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean -4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean -4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval -1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile.
DISCUSSION: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:64 |
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| Enthalten in: |
Muscle & nerve - 64(2021), 6 vom: 29. Dez., Seite 662-669 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Siddiqi, Zaeem A [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 18.03.2022 Date Revised 31.05.2022 published: Print-Electronic ClinicalTrials.gov: NCT01997229, NCT02301624 Citation Status MEDLINE |
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| doi: |
10.1002/mus.27422 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM331321912 |
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| 100 | 1 | |a Siddiqi, Zaeem A |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab |b subgroup analysis of REGAIN and its extension study |
| 264 | 1 | |c 2021 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 18.03.2022 | ||
| 500 | |a Date Revised 31.05.2022 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a ClinicalTrials.gov: NCT01997229, NCT02301624 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2021 Wiley Periodicals LLC. | ||
| 520 | |a INTRODUCTION/AIMS: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+ ) gMG previously treated with rituximab | ||
| 520 | |a METHODS: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE | ||
| 520 | |a RESULTS: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean -4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean -4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval -1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile | ||
| 520 | |a DISCUSSION: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously | ||
| 650 | 4 | |a Clinical Trial, Phase III | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a acetylcholine receptor | |
| 650 | 4 | |a eculizumab | |
| 650 | 4 | |a myasthenia gravis | |
| 650 | 4 | |a refractory | |
| 650 | 4 | |a rituximab | |
| 650 | 7 | |a Antibodies, Monoclonal, Humanized |2 NLM | |
| 650 | 7 | |a Rituximab |2 NLM | |
| 650 | 7 | |a 4F4X42SYQ6 |2 NLM | |
| 650 | 7 | |a eculizumab |2 NLM | |
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| 700 | 1 | |a Nowak, Richard J |e verfasserin |4 aut | |
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| 700 | 1 | |a Yountz, Marcus |e verfasserin |4 aut | |
| 700 | 1 | |a Patti, Francesco |e verfasserin |4 aut | |
| 700 | 0 | |a REGAIN Study Group |e verfasserin |4 aut | |
| 700 | 1 | |a Mazia, Claudio Gabriel |e investigator |4 oth | |
| 700 | 1 | |a Wilken, Miguel |e investigator |4 oth | |
| 700 | 1 | |a Wilken, Miguel |e investigator |4 oth | |
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