ABSTRACT
Myasthenia gravis (MG) is an autoimmune disease characterized by fluctuating weakness of skeletal muscles. Despite current treatments, a significant percentage of patients remain symptomatic. This review explores new immunosuppressive therapies and ongoing clinical trials in MG, including depletion of B lymphocytes with agents such as rituximab and inebilizumab, as well as the use of eculizumab, efgartigimod, satralizumab, tocilizumab, and CAR-T (Chimeric Antigen Receptor-T) cell therapy. These advancements aim to improve disease control and patients' quality of life.
La myasthénie grave (MG) est une maladie auto-immune caractérisée par une faiblesse fluctuante des muscles squelettiques. Malgré les traitements classiques, un pourcentage significatif de patients reste symptomatique. Cet article explore les nouvelles thérapies immunosuppressives et les essais cliniques en cours pour la MG, notamment la déplétion des lymphocytes B avec des agents tels que le rituximab et l'inébilizumab, ainsi que l'utilisation de l'éculizumab, de l'efgartigimod, du satralizumab, du tocilizumab et de la thérapie par cellules CAR-T (Chimeric Antigen Receptor-T). Ces avancées visent à améliorer le contrôle de la maladie et la qualité de vie des patients.
Subject(s)
Myasthenia Gravis , Myasthenia Gravis/immunology , Myasthenia Gravis/therapy , Myasthenia Gravis/drug therapy , Humans , Immunosuppressive Agents/therapeutic use , Quality of Life , Immunomodulating Agents/therapeutic use , B-Lymphocytes/immunology , B-Lymphocytes/drug effects , Immunologic Factors/therapeutic useABSTRACT
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is the most common chronic autoimmune neuropathy. Its management has considerably evolved over the last decade. In 2021, the diagnostic guidelines for CIDP were updated and the diagnostic criteria simplified. They enable better characterization of the electro-clinical phenotype of the disease, and emphasize supportive criteria, in particular neuro-muscular imaging. In terms of pathophysiology, the discovery of antibodies directed against antigens in the nodal and paranodal regions has given rise to the concept of autoimmune nodopathy. Finally, the preliminary results of the ADHERE study on efgartigimod have rekindled hopes of a new, effective therapy for CIDP.
La polyradiculoneuropathie inflammatoire démyélinisante chronique (PIDC) est la neuropathie auto-immune chronique la plus fréquente. Sa prise en charge a largement évolué durant la dernière décennie. En 2021, les recommandations diagnostiques de la PIDC ont été mises à jour et les critères diagnostiques simplifiés. Ils permettent une meilleure caractérisation du phénotype électroclinique de la maladie et mettent en avant les critères de support diagnostiques, en particulier l'imagerie neuromusculaire. Sur le plan physiopathologique, la découverte d'anticorps dirigés contre des antigènes des régions nodale et paranodale a fait naître le concept de nodopathie auto-immune. Enfin, les résultats préliminaires de l'étude ADHERE sur l'efgartigimod font émerger l'espoir d'une nouvelle thérapie efficace dans la PIDC.
Subject(s)
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating , Humans , Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/diagnosis , Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/therapyABSTRACT
INTRODUCTION: To describe the clinical characteristics and long term outcome of CIDP patients according to 2021 EAN/PNS diagnostic certainty categories. METHODS: We reviewed clinical data, response to treatment, cerebrospinal fluid examination, and nerve conduction studies parameters of 39 adult "CIDP" and 24 "possible CIDP" patients. Data were collected at diagnosis and after one (T1), two (T2), three (T3) and five years (T5). RESULTS: At diagnosis, "possible CIDP" patients' phenotypes were more atypical (especially focal/multifocal, p < .01) and "CIDP" patients had a higher NIS and INCAT scores (p = .08 and 0.08). Compared to baseline: median NIS score decreased in "CIDP" and was stable in "possible CIDP" patients at T1 (p < .05), T2 (p < .05) and T3 (p < .01); median MRC score slightly increased in "CIDP" and was stable in "possible CIDP" patients at T2 (p < .05); and INCAT disability scale slightly decreased in "CIDP" and was stable in "possible CIDP" patients at T3 (p < .05). The proportion of moderate to severely disabled (mRS > 2) patients in "possible CIDP" group was higher than in "CIDP" group (not significant). "CIDP" patients had a better objective response to immunotherapy (59 % responders) than "possible CIDP" patients (29 % responders, p < .05), especially among typical CIDP patients (86 % of responders in "CIDP" versus 33 % of responders in "possible CIDP" patients, p < .05). CONCLUSION: "CIDP" patients had a more severe neuropathy, estimated with the NIS and INCAT scores, and "possible CIDP" patients had a more atypical phenotype at baseline. Our data suggest that long-term patient outcome and response to immunotherapy is better in "CIDP" than "possible CIDP".