Drug-refractory myasthenia gravis : Clinical characteristics, treatments, and outcome
Cortés-Vicente, Elena (Universitat Autònoma de Barcelona. Departament de Medicina)
Álvarez-Velasco, Rodrigo (Institut d'Investigació Biomèdica Sant Pau)
Pla-Junca, Francesc (Instituto de Salud Carlos III)
Rojas-Garcia, Ricard (Institut d'Investigació Biomèdica Sant Pau)
Paradas, Carmen (Instituto de Biomedicina de Sevilla)
Sevilla, Teresa (Universitat de València. Departament de Medicina)
Casasnovas, Carlos (Hospital Universitari de Bellvitge)
Gómez-Caravaca, María Teresa (Hospital Universitario Reina Sofía (Còrdova, Espanya))
Pardo, Julio (Hospital Clínico Universitario (Santiago de Compostela, Galícia))
Ramos-Fransi, Alba (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol)
Pelayo-Negro, Ana Lara (Universidad de Cantabria)
Gutiérrez-Gutiérrez, Gerardo (Universidad Europea de Madrid)
Turon-Sans, Janina (Universitat Autònoma de Barcelona. Departament de Medicina)
López de Munain, Adolfo (Biodonostia Osasun Ikerketako Institutura (País Basc))
Guerrero-Sola, Antonio (Hospital Clínico San Carlos (Madrid))
Jericó-Pascual, Ivonne (Instituto de Investigación Sanitaria de Navarra)
Martín, María Asunción (Hospital General Yagüe (Burgos))
Mendoza, María Dolores (Hospital Universitario de Gran Canaria Dr. Negrín)
Morís, Germán (Hospital Universitario Central de Asturias)
Vélez-Gómez, Beatriz (Universidad de Sevilla)
García-Sobrino, Tania (Hospital Clínico Universitario (Santiago de Compostela, Galícia))
Pascual-Goñi, Elba (Institut d'Investigació Biomèdica Sant Pau)
Reyes-Leiva, David (Institut d'Investigació Biomèdica Sant Pau)
Illa, Isabel (Institut d'Investigació Biomèdica Sant Pau)
Gallardo, Eduard (Institut d'Investigació Biomèdica Sant Pau)

Date:	2022
Abstract:	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. 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. 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. In this study, 8. 5% of MG patients were drug-refractory. New more specific drugs are needed to treat drug-refractory MG patients.
Grants:	Instituto de Salud Carlos III JR19/00037 Instituto de Salud Carlos III PI19/01774
Note:	Altres ajuts: R. Alvarez-Velasco was supported by grant SLT008/18/00207 from the Health Research and Innovation Strategic Plan (PERIS). The NMD-ES Project and F. PlaJunca (data curator) are partially funded by the Centro de Investigacion Biomédica en Red de Enfermedades Raras (CIBERER).
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Published in:	Annals of Clinical and Translational Neurology, Vol. 9 Núm. 2 (january 2022) , p. 122-131, ISSN 2328-9503

DOI: 10.1002/acn3.51492
PMID: 35080153

10 p, 758.5 KB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP)
Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut de Recerca Sant Pau
Articles > Research articles
Articles > Published articles