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. 2023 Dec 8:16:5835-5843.
doi: 10.2147/JIR.S423098. eCollection 2023.

A Pilot Study on Tocilizumab in Very-Late-Onset Myasthenia Gravis

Ting-Ting Yang #  1 , Ze-Yi Wang #  1 , Ze-Xin Fan  1 , Bo-Yi Yuan  1 , Lin Ma  1 , Jian-Feng Lu  1 , Pen-Ju Liu  1 , Yang He  2 , Guang-Zhi Liu  1
Affiliations

A Pilot Study on Tocilizumab in Very-Late-Onset Myasthenia Gravis

Ting-Ting Yang et al. J Inflamm Res. .

Abstract

Objective: This study aimed to initially investigate the efficacy and safety of low-dose tocilizumab combined with glucocorticoid for the treatment of very-late-onset myasthenia gravis (VLOMG).

Methods: We conducted a retrospective study in VLOMG patients who were administered intravenous methylprednisolone therapy and subsequently received low-dose oral corticosteroid, in combination with intravenous injection of tocilizumab given once every month for three months.

Results: Five patients (mean age 75.0 ± 4.5 years) were included, and all of them were new-onset, and anti-acetylcholine receptor (AChR) antibody-positive generalized MG. The Quantitative Myasthenia Gravis Scale (QMGS) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scores before treatment were 15.4 ± 4.3 and 9.6 ± 2.3, respectively, and they exhibited a continuously decreasing trend after the first, second, and third injection of tocilizumab until 6 months after treatment. At 6 months post-treatment, the QMGS and MG-ADL scores were 5.0 ± 2.9 and 2.0 ± 1.2, respectively, and the difference between scores at baseline and 6-month follow-up was significant (P = 0.005 and P < 0.0001, respectively). No serious adverse drug reactions were reported in any patient during the study period.

Discussions and conclusion: The therapeutic efficacy of tocilizumab in VLOMG remains uncertain. The results from our study support the efficacy and safety of this combination treatment option for VLOMG, and strongly suggests the therapeutic potential of tocilizumab in VLOMG. However, considering the limitation of retrospective nature and small sample size in this study, prospective randomized controlled studies including a larger sample size of selected patients are needed to validate our results.

Keywords: glucocorticoid; immunotherapy; tocilizumab; very late-onset myasthenia gravis.

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Conflict of interest statement

The authors declare that they have no conflicts of interest in this work.

Figures

Figure 1
Figure 1
Clinical outcomes in patients with very-late-onset myasthenia gravis (VLOMG). Dynamic changes in (A) the Myasthenia Gravis Activities of Daily Living (MG-ADL) score and (B) Quantitative Myasthenia Gravis Scale (QMGS) score in five patients before treatment (baseline) and at the time of the first, second, and third doses of tocilizumab, as well as at 6 months after treatment. *P < 0.05, **P < 0.01, ***P < 0.001, ****P < 0.0001.
Figure 2
Figure 2
Changes in serum anti-acetylcholine receptor (AChR) antibody titers were observed in two patients with very-late-onset myasthenia gravis before treatment (baseline) and at the time of the first, second, and third doses of tocilizumab.
See this image and copyright information in PMC

References

    1. Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009;8(5):475–490. doi:10.1016/S1474-4422(09)70063-8 - DOI - PMC - PubMed
    1. Lascano AM, Lalive PH. Update in immunosuppressive therapy of myasthenia gravis. Autoimmun Rev. 2021;20(1):102712. doi:10.1016/j.autrev.2020.102712 - DOI - PubMed
    1. Gilhus NE. Myasthenia Gravis. N Engl J Med. 2016;375(26):2570–2581. doi:10.1056/NEJMra1602678 - DOI - PubMed
    1. Barnett C, Bril V. New insights into very-late-onset myasthenia gravis. Nat Rev Neuro. 2020;16(6):299–300. doi:10.1038/s41582-020-0345-3 - DOI - PubMed
    1. Cortés-Vicente E, Álvarez-velasco R, Segovia S, et al. Clinical and therapeutic features of myasthenia gravis in adults based on age at onset. Neurology. 2020;94(11):e1171–e1180. doi:10.1212/WNL.0000000000008903 - DOI - PMC - PubMed