Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome
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...
| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2022-02-01
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| Series: | Annals of Clinical and Translational Neurology |
| Online Access: | https://doi.org/10.1002/acn3.51492 |
| _version_ | 1818984984602476544 |
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| author | Elena Cortés‐Vicente Rodrigo Álvarez‐Velasco Francesc Pla‐Junca Ricard Rojas‐Garcia Carmen Paradas Teresa Sevilla Carlos Casasnovas María Teresa Gómez‐Caravaca Julio Pardo Alba Ramos‐Fransi Ana Lara Pelayo‐Negro Gerardo Gutiérrez‐Gutiérrez Janina Turon‐Sans Adolfo López de Munain Antonio Guerrero‐Sola Ivonne Jericó María Asunción Martín María Dolores Mendoza Germán Morís Beatriz Vélez‐Gómez Tania Garcia‐Sobrino Elba Pascual‐Goñi David Reyes‐Leiva Isabel Illa Eduard Gallardo |
| author_facet | Elena Cortés‐Vicente Rodrigo Álvarez‐Velasco Francesc Pla‐Junca Ricard Rojas‐Garcia Carmen Paradas Teresa Sevilla Carlos Casasnovas María Teresa Gómez‐Caravaca Julio Pardo Alba Ramos‐Fransi Ana Lara Pelayo‐Negro Gerardo Gutiérrez‐Gutiérrez Janina Turon‐Sans Adolfo López de Munain Antonio Guerrero‐Sola Ivonne Jericó María Asunción Martín María Dolores Mendoza Germán Morís Beatriz Vélez‐Gómez Tania Garcia‐Sobrino Elba Pascual‐Goñi David Reyes‐Leiva Isabel Illa Eduard Gallardo |
| author_sort | Elena Cortés‐Vicente |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-20T18:27:41Z |
| format | Article |
| id | doaj.art-2b39a018049e4bdbb38862338fe7dbf0 |
| institution | Directory Open Access Journal |
| issn | 2328-9503 |
| language | English |
| last_indexed | 2024-12-20T18:27:41Z |
| publishDate | 2022-02-01 |
| publisher | Wiley |
| record_format | Article |
| series | Annals of Clinical and Translational Neurology |
| spelling | doaj.art-2b39a018049e4bdbb38862338fe7dbf02022-12-21T19:30:06ZengWileyAnnals of Clinical and Translational Neurology2328-95032022-02-019212213110.1002/acn3.51492Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcomeElena Cortés‐Vicente0Rodrigo Álvarez‐Velasco1Francesc Pla‐Junca2Ricard Rojas‐Garcia3Carmen Paradas4Teresa Sevilla5Carlos Casasnovas6María Teresa Gómez‐Caravaca7Julio Pardo8Alba Ramos‐Fransi9Ana Lara Pelayo‐Negro10Gerardo Gutiérrez‐Gutiérrez11Janina Turon‐Sans12Adolfo López de Munain13Antonio Guerrero‐Sola14Ivonne Jericó15María Asunción Martín16María Dolores Mendoza17Germán Morís18Beatriz Vélez‐Gómez19Tania Garcia‐Sobrino20Elba Pascual‐Goñi21David Reyes‐Leiva22Isabel Illa23Eduard Gallardo24Neuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) Instituto de Salud Carlos III Madrid SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainNeurology Department, Neuromuscular Disorders Unit, Instituto de Biomedicina de Sevilla, Hospital U. Virgen del Rocío, CSIC Universidad de Sevilla Sevilla SpainCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) Instituto de Salud Carlos III Madrid SpainCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) Instituto de Salud Carlos III Madrid SpainNeurology Department Hospital Universitario Reina Sofía Córdoba SpainNeurology Department Hospital Clínico Santiago de Compostela SpainNeuromuscular Diseases Unit, Department of Neurology Hospital Germans Trias i Pujol Badalona, Barcelona SpainCentro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED) Instituto de Salud Carlos III Madrid SpainNeuromuscular Diseases Unit, Department of Neurology Hospital Universitario Infanta Sofía, Universidad Europea de Madrid San Sebastián de los Reyes, Madrid SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainCentro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED) Instituto de Salud Carlos III Madrid SpainNeuromuscular Diseases Unit, Department of Neurology, Institute of Neurosciences Hospital Universitario Clínico San Carlos Madrid SpainDepartment of Neurology Complejo Hospitalario de Navarra‐IdisNA Pamplona SpainDepartment of Neurology Complejo Asistencial Hospitalario de Burgos Burgos SpainDepartment of Neurology Hospital Universitario de Gran Canaria Doctor Negrín Las Palmas de Gran Canaria SpainDepartment of Neurology Hospital Central de Asturias Oviedo SpainNeurology Department, Neuromuscular Disorders Unit, Instituto de Biomedicina de Sevilla, Hospital U. Virgen del Rocío, CSIC Universidad de Sevilla Sevilla SpainNeurology Department Hospital Clínico Santiago de Compostela SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainNeuromuscular Diseases Unit, Department of Neurology Hospital de la Santa Creu i Sant Pau Barcelona SpainAbstract 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.https://doi.org/10.1002/acn3.51492 |
| spellingShingle | Elena Cortés‐Vicente Rodrigo Álvarez‐Velasco Francesc Pla‐Junca Ricard Rojas‐Garcia Carmen Paradas Teresa Sevilla Carlos Casasnovas María Teresa Gómez‐Caravaca Julio Pardo Alba Ramos‐Fransi Ana Lara Pelayo‐Negro Gerardo Gutiérrez‐Gutiérrez Janina Turon‐Sans Adolfo López de Munain Antonio Guerrero‐Sola Ivonne Jericó María Asunción Martín María Dolores Mendoza Germán Morís Beatriz Vélez‐Gómez Tania Garcia‐Sobrino Elba Pascual‐Goñi David Reyes‐Leiva Isabel Illa Eduard Gallardo Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome Annals of Clinical and Translational Neurology |
| title | Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
| title_full | Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
| title_fullStr | Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
| title_full_unstemmed | Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
| title_short | Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome |
| title_sort | drug refractory myasthenia gravis clinical characteristics treatments and outcome |
| url | https://doi.org/10.1002/acn3.51492 |
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