Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Jun;94(6):467-473.
doi: 10.1136/jnnp-2022-330519. Epub 2023 Jan 24.

Effectiveness of early cycles of fast-acting treatment in generalised myasthenia gravis

Akiyuki Uzawa  1 , Shigeaki Suzuki  2 , Satoshi Kuwabara  3 , Hiroyuki Akamine  3 , Yosuke Onishi  3 , Manato Yasuda  3 , Yukiko Ozawa  3 , Naoki Kawaguchi  4 , Tomoya Kubota  5 , Masanori P Takahashi  5 , Yasushi Suzuki  6 , Genya Watanabe  6 , Takashi Kimura  7 , Takamichi Sugimoto  8 , Makoto Samukawa  9 , Naoya Minami  10 , Masayuki Masuda  11 , Shingo Konno  12 , Yuriko Nagane  13 , Kimiaki Utsugisawa  13
Affiliations

Effectiveness of early cycles of fast-acting treatment in generalised myasthenia gravis

Akiyuki Uzawa et al. J Neurol Neurosurg Psychiatry. 2023 Jun.

Abstract

Background: Early fast-acting treatment (EFT) is the aggressive use of fast-acting therapies such as plasmapheresis, intravenous immunoglobulin and/or intravenous high-dose methylprednisolone (IVMP) from the early phases of treatment. EFT is reportedly beneficial for early achievement of minimal manifestations (MM) or better status with ≤5 mg/day prednisolone (MM5mg), a practical therapeutic target for myasthenia gravis (MG).

Objective: The current study aimed to clarify which specific EFT regimen is efficacious and the patient characteristics that confer sensitivity to EFT.

Methods: We recruited a total of 1710 consecutive patients with MG who enrolled in the Japan MG Registry for this large-cohort study. Among them, 1066 with generalised MG who had received immunotherapy were analysed. Prognostic background factors were matched in a 1:1 ratio using propensity score matching analysis between patients treated with EFT (n=350) and those treated without EFT (n=350). The clinical course and time to first achieve MM5mg after starting immunotherapy was analysed in relation to treatment combinations and patient characteristics.

Results: Kaplan-Meier analyses showed that EFT had a significant effect on the achievement of MM5mg (p<0.0001, log-rank test; HR 1.82, p<0.0001). Notably, EFT was efficacious for any type of MG, and the inclusion of IVMP resulted in earlier and more frequent achievement of MM5mg (p=0.0352, log-rank test; HR 1.46, p=0.0380). In addition, early administration of calcineurin inhibitors also promoted MM5mg achievement.

Conclusion: Early cycles of intervention with EFT and early use of calcineurin inhibitors provides long-term benefits in terms of achieving therapeutic targets for generalised MG, regardless of clinical subtype.

Keywords: MYASTHENIA; NEUROIMMUNOLOGY; NEUROMUSCULAR.

PubMed Disclaimer

Conflict of interest statement

Competing interests: AU has received honoraria from Alexion Pharmaceuticals and Argenx. SS received speakers’ fees from Alexion Pharmaceuticals, the Japan Blood Products Organization, Asahi Kasei Medical and Argenx and participated on advisory board meeting of Alexion Pharmaceuticals and Argenx. SK has received Research Grant on Intractable Diseases (Neuroimmunological Diseases) from the Ministry of Health, Labour and Welfare of Japan (20FC1030) and honoraria from CSL Behring. TK reports honorarium for lectures from Alexon Pharmaceuticals. MPT reports unrestricted research grants from Japan Blood Products Organization, Astellas Pharma, Mitsubishi Tanabe Pharma and Pfizer, outside the submitted work, and has served as a paid Consultant for Alexion, Argenx, Sanofi, and UCB Pharma and received honorarium for lectures from Argenx, Alexion Pharmaceuticals and UCB Pharma. MM has received speaker honoraria from Argenx, Asahi Kasei Medical, Alexion Pharmaceutical and participated on advisory board meeting of Alexion Pharmaceutical. KU has served as a paid Consultant for UCB Pharma, Janssen Pharma, Horizon Therpeutics (Viela Bio), Chugai Pharma, Hanall BioPharma and Mitsubishi Tanabe Pharma, and has received speaker honoraria from Argenx, Alexion Pharmaceuticals, UCB Pharma, and the Japan Blood Products Organisation. Other authors declare no financial or other conflicts of interest.

Publication types

LinkOut - more resources