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. 2024 May;11(5):1338-1346.
doi: 10.1002/acn3.52051. Epub 2024 Apr 4.

Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis

Daiki Tokuyasu  1 , Shigeaki Suzuki  1 , Akiyuki Uzawa  2 , Yuriko Nagane  3 , Masayuki Masuda  4 , Shingo Konno  5 , Tomoya Kubota  6 , Makoto Samukawa  7 , Takamichi Sugimoto  8 , Kei Ishizuchi  1 , Munenori Oyama  1 , Manato Yasuda  2 , Hiroyuki Akamine  2 , Yosuke Onishi  2 , Yasushi Suzuki  9 , Naoki Kawaguchi  10 , Naoya Minami  11 , Takashi Kimura  12 , Masanori P Takahashi  6 , Hiroyuki Murai  13 , Kimiaki Utsugisawa  3
Affiliations

Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis

Daiki Tokuyasu et al. Ann Clin Transl Neurol. 2024 May.

Abstract

Objective: Eculizumab and ravulizumab are complement protein C5 inhibitors, showing efficacy and tolerability for patients with anti-acetylcholine receptor-positive (AChR+) generalized myasthenia gravis (gMG) in phase 3 clinical trials and subsequent analyses. The purpose of the present study was to evaluate the clinical significance of eculizumab and switching to ravulizumab for refractory AChR+ gMG patients in the real-world experience.

Methods: Among the database of Japan MG registry survey 2021, we studied AChR+ gMG patients who received eculizumab. We also evaluated these patients who switched from eculizumab to ravulizumab. Responder was defined as an improvement of at least 3 points in MG-ADL. We performed a questionnaire of preference between eculizumab and ravulizumab.

Results: Among 1,106 patients with AChR+ gMG, 36 patients (3%) received eculizumab (female 78%, mean age 56.0 years). Eculizumab was preferentially used in severe and refractory MG patients. The duration of eculizumab treatment was 35 months on average. MG-ADL improved from 9.4 ± 4.9 to 5.9 ± 5.1, and 25 (70%) of the 36 gMG patients were responders. Postintervention status was markedly improved after the eculizumab treatment. Of 13 patients who did not continue eculizumab, 6 showed insufficiencies. Early onset MG was most effective. However, 15 patients switching from eculizumab to ravulizumab kept favorable response and tolerability. Questionnaire surveys showed preference for ravulizumab over eculizumab.

Interpretation: Eculizumab and switching to ravulizumab showed to be effective for refractory AChR+ gMG patients in clinical settings.

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Conflict of interest statement

D. Tokuyasu declares no potential conflicts of interest related to this article; S. Suzuki received personal fees from Alexion Pharmaceuticals, Argenx, and UCB Pharma, the Japan Blood Products Organization, and Asahi Kasei Medical; A. Uzawa has received honoraria from Alexion Pharmaceuticals and Argenx; Y. Nagane has received speaker honoraria from argenx, Alexion Pharmaceuticals and Japan Blood Products Organization; M. Masuda declares no potential conflicts of interest related to this article; S. Konno declares no potential conflicts of interest related to this article; T. Kubota declares receiving an honorarium for lectures from Alexon Pharmaceuticals, Argenx, and UCB Pharma; M. Samukawa declares no potential conflicts of interest related to this article; T. Sugimoto declares no potential conflicts of interest related to this article; K. Ishizuchi declares no potential conflicts of interest related to this article; M. Oyama declares no potential conflicts of interest related to this article; M. Yasuda declares no potential conflicts of interest related to this article; H. Akamine declares no potential conflicts of interest related to this article; Y. Onishi declares no potential conflicts of interest related to this article; Y. Suzuki declares no potential conflicts of interest related to this article; N. Kawaguchi declares no potential conflicts of interest related to this article; N. Minami declares no potential conflicts of interest related to this article; T. Kimura declares no potential conflicts of interest related to this article; M. P. Takahashi reports unrestricted research grants from Japan Blood Products Organization, Astellas Pharma, Mitsubishi Tanabe Pharma and Pfizer, outside the submitted work, and honorarium for lectures from argenx, Alexion Pharmaceuticals and UCB Pharma; H. Murai has served as a paid consultant for Alexion, AstraZeneca Rare Disease, Argenx, and UCB and has received speaker honoraria from the Japan Blood Products Organization and Chugai Pharmaceutical, and research support from the Ministry of Health, Labour and Welfare, Japan; K. Utsugisawa has served as a paid consultant for UCB Pharma, Argenx, Janssen Pharma, Viela Bio, Chugai Pharma, Hanall BioPharma, and Mitsubishi Tanabe Pharma and has received speaker honoraria from argenx, Alexion Pharmaceuticals, and the Japan Blood Products Organization.

Figures

Figure 1
Figure 1
Study flow. AChR+: anti‐acetylcholine receptor‐positive; MG, myasthenia gravis.
Figure 2
Figure 2
Changes in the mean MG‐ADL (A) and the mean daily prednisolone (B). MG‐ADL, myasthenia gravis activity of daily living.
Figure 3
Figure 3
Outcome of patients. Changes in the patients' postintervention status. I, improved; MM, minimal manifestations; U, unchanged; W, worse.
Figure 4
Figure 4
Questionnaire of preferences between eculizumab and ravulizumab.
See this image and copyright information in PMC

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