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Title: | Drug-refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
Author: | Cortés Vicente, Elena Álvarez-Velasco, Rodrigo Pla-Junca, Francesc Rojas-Garcia, Ricard Paradas, Carmen Sevilla, Teresa Casasnovas, Carlos Gómez-Caravaca, María Teresa Pardo, Julio Ramos-Fransi, Alba Pelayo-Negro, Ana Lara Gutiérrez-Gutiérrez, Gerardo Turon Sans, Janina López de Munain, Adolfo Guerrero Sola, Antonio Jericó, Ivonne Martín, María Asunción Mendoza, María Dolores Morís, Germán Vélez-Gómez, Beatriz García-Sobrino, Tania Pascual-Goñi, Elba Reyes-Leiva, David Illa Sendra, Isabel Gallardo, Eduard |
Keywords: | Assaigs clínics de medicaments Malalties musculars Immunologia Efectes secundaris dels medicaments Drug testing Muscular Diseases Immunology Drug side effects |
Issue Date: | Jan-2022 |
Publisher: | American Neurological Association |
Abstract: | Objective: To describe the clinical characteristics and outcomes in patients with refractory myasthenia gravis (MG) and to determine the effectiveness and side effects of the drugs used for their treatment. Methods: This observational retrospective cross-sectional multicenter study was based on data from the Spanish MG Registry (NMD-ES). Patients were considered refractory when their MG Foundation of America post-interventional status (MGFA-PIS) was unchanged or worse after corticosteroids and two or more other immunosuppressive agents. Clinical and immunologic characteristics of drug-refractory patients, efficiency and toxicity of drugs used, and outcome (MGFA-PIS) at end of follow-up were studied. Results: We included 990 patients from 15 hospitals. Eighty-four patients (68 of 842 anti-acetylcholine receptor [AChR], 5 of 26 anti-muscle-specific tyrosine kinase [MusK], 10 of 120 seronegative, and 1 of 2 double-seropositive patients) were drug refractory. Drug-refractory patients were more frequently women (p < 0.0001), younger at onset (p < 0.0001), and anti-MuSK positive (p = 0.037). Moreover, they more frequently presented a generalized form of the disease, bulbar symptoms, and life-threatening events (p < 0.0001; p = 0.018; and p = 0.002, respectively) than non-drug-refractory patients. Mean follow-up was 9.8 years (SD 4.5). Twenty-four (50%) refractory patients had side effects to one or more of the drugs. At the end of follow-up, 42.9% of drug-refractory patients (42.6% of anti-AChR, 100% of anti-MuSK, and 10% of seronegative patients) and 79.8% of non-drug-refractory patients (p < 0.0001) achieved remission or had minimal manifestations. Eighty percent of drug-refractory-seronegative patients did not respond to any drug tested. Interpretation: In this study, 8.5% of MG patients were drug-refractory. New more specific drugs are needed to treat drug-refractory MG patients. |
Note: | Reproducció del document publicat a: https://doi.org/10.1002/acn3.51492 |
It is part of: | Annals of Clinical and Translational Neurology, 2022, vol. 9, num. 2, p. 122-131 |
URI: | http://hdl.handle.net/2445/198171 |
Related resource: | https://doi.org/10.1002/acn3.51492 |
ISSN: | 2328-9503 |
Appears in Collections: | Articles publicats en revistes (Ciències Clíniques) |
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