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Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis.
Tokuyasu, Daiki; Suzuki, Shigeaki; Uzawa, Akiyuki; Nagane, Yuriko; Masuda, Masayuki; Konno, Shingo; Kubota, Tomoya; Samukawa, Makoto; Sugimoto, Takamichi; Ishizuchi, Kei; Oyama, Munenori; Yasuda, Manato; Akamine, Hiroyuki; Onishi, Yosuke; Suzuki, Yasushi; Kawaguchi, Naoki; Minami, Naoya; Kimura, Takashi; Takahashi, Masanori P; Murai, Hiroyuki; Utsugisawa, Kimiaki.
Afiliación
  • Tokuyasu D; Department of Neurology, Keio University School of Medicine, Tokyo, Japan.
  • Suzuki S; Department of Neurology, Keio University School of Medicine, Tokyo, Japan.
  • Uzawa A; Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Nagane Y; Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
  • Masuda M; Department of Neurology, Tokyo Medical University, Tokyo, Japan.
  • Konno S; Department of Neurology, Toho University Ohashi Medical Center, Tokyo, Japan.
  • Kubota T; Department of Clinical Laboratory and Biomedical Sciences, Division of Health Sciences, Osaka University Graduate School of Medicine, Osaka, Japan.
  • Samukawa M; Department of Neurology, Kindai University Faculty of Medicine, Sayama, Japan.
  • Sugimoto T; Department of Clinical Neuroscience and Therapeutics, Hiroshima University, Hiroshima, Japan.
  • Ishizuchi K; Department of Neurology, Keio University School of Medicine, Tokyo, Japan.
  • Oyama M; Department of Neurology, Keio University School of Medicine, Tokyo, Japan.
  • Yasuda M; Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Akamine H; Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Onishi Y; Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Suzuki Y; Department of Neurology, National Hospital Organization Sendai Medical Center, Sendai, Japan.
  • Kawaguchi N; Department of Neurology, Neurology Chiba Clinic, Chiba, Japan.
  • Minami N; Department of Neurology, National Hospital Organization Hokkaido Medical Center, Sapporo, Japan.
  • Kimura T; Department of Neurology, Hyogo Medical University, Nishinomiya, Japan.
  • Takahashi MP; Department of Clinical Laboratory and Biomedical Sciences, Division of Health Sciences, Osaka University Graduate School of Medicine, Osaka, Japan.
  • Murai H; Department of Neurology, International University of Health and Welfare, Narita, Japan.
  • Utsugisawa K; Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
Ann Clin Transl Neurol ; 11(5): 1338-1346, 2024 May.
Article en En | MEDLINE | ID: mdl-38572524
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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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inactivadores del Complemento / Anticuerpos Monoclonales Humanizados / Miastenia Gravis Límite: Adult / Aged / Female / Humans / Male / Middle aged País/Región como asunto: Asia Idioma: En Revista: Ann Clin Transl Neurol Año: 2024 Tipo del documento: Article País de afiliación: Japón Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inactivadores del Complemento / Anticuerpos Monoclonales Humanizados / Miastenia Gravis Límite: Adult / Aged / Female / Humans / Male / Middle aged País/Región como asunto: Asia Idioma: En Revista: Ann Clin Transl Neurol Año: 2024 Tipo del documento: Article País de afiliación: Japón Pais de publicación: Estados Unidos