Rinsho Shinkeigaku (Clinical Neurology)

Original Article

Usefulness of an anti-mouse cerebellar tissue-derived antigen antibody test in predicting immunotherapy efficacy in patients with idiopathic cerebellar ataxia

Takayuki Ohtomo, Ph.D.1), Sakura Ogino1), Sayaka Yanai1), Sayaka Nakayama1), Risa Yajima1), Mayu Sugawara1), Junji Yamada, Ph.D.1), Hitoshi Aizawa, M.D., Ph.D.2), Takeshi Taguchi, M.D.3) and Kazunori Nanri, M.D., Ph.D.3)4)

1) Center for Clinical Pharmacy, Tokyo University of Pharmacy and Life Sciences
2) Department of Neurology, Tokyo Medical University
3) Department of Neurology, Tokyo Medical University Hachioji Medical Center
4) Department of Internal Medicine, Hanno Geriatirc Center

Background: Autoimmune cerebellar ataxia (AICA) is a general term for diseases in which the cerebellum is damaged by an autoimmune mechanism. For the diagnosis of the AICA, anti-thyroid antibodies (anti-thyroid peroxidase antibody and anti-thyroglobulin antibody), anti-glutamic acid decarboxylase (GAD) antibodies, and anti-gliadin antibodies are measured. Immunotherapy is known to be effective for AICA, but some patients with effective immunotherapy lack autoantibodies associated with cerebellar ataxia. The purpose of this study was to clarify whether the effectiveness of immunotherapy in patients with suspected AICA could be predicted by anti-mouse cerebellar tissue-derived antigen antibody tests. Methods: This study was conducted on 25 patients with idiopathic cerebellar ataxia (excluding multiple system atrophy, hereditary spinocerebellar degeneration, cancer-bearing patients, and patients taking phenytoin) who received immunotherapy from 2005 to 2016 at Tokyo Medical University Hachioji Medical Center. The patients were suspected of having AICA because they were positive for cerebellar ataxia-related autoantibodies (anti-thyroid antibody, anti-GAD antibody, anti-gliadin antibody, or anti-transglutaminase 6 antibody) or other autoantibodies. Antibodies that bind to mouse cerebellar tissue-derived antigens were defined as "anti-mouse cerebellar tissue-derived antigen antibodies" in this study, and their IgG-class antibodies were comprehensively measured using a slot blot. Results: Antimouse cerebellar tissue-derived antigen antibody test results were correlated with immunotherapy efficacy. Furthermore, the combination of anti-mouse cerebellar tissue-derived antigen and anti-GAD antibody tests could predict the effectiveness of immunotherapy with 83% sensitivity and 100% specificity, while the combination of the anti-mouse cerebellar tissue-derived antigen, anti-GAD, and anti-gliadin (IgA class) antibody tests could predict the effectiveness of immunotherapy with 94% sensitivity and 86% specificity. Conclusion: Anti-mouse cerebellar tissue-derived antigen antibody tests could help to provide useful information for immunotherapy administration to patients with idiopathic cerebellar ataxia suspected to be AICA.
Full Text of this Article in Japanese PDF (1882K)

(CLINICA NEUROL, 62: 112−122, 2022)
key words: Idiopathic cerebellar ataxia, autoimmune cerebellar ataxia, primary autoimmune cerebellar ataxia, anti-mouse cerebellar tissue-derived antigen antibodies, immunotherapy

(Received: 25-Mar-21)