Rituximab as Adjunct Maintenance Therapy for Refractory Juvenile Myasthenia Gravis
| Autor: | Peter B. Kang, Melissa E. Elder, Carla D. Zingariello |
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| Rok vydání: | 2020 |
| Předmět: |
Male
Pediatrics medicine.medical_specialty Adolescent 03 medical and health sciences 0302 clinical medicine Developmental Neuroscience Maintenance therapy immune system diseases 030225 pediatrics Myasthenia Gravis Outcome Assessment Health Care Humans Immunologic Factors Medicine Receptors Cholinergic Child Adverse effect Autoantibodies Retrospective Studies business.industry Receptor Protein-Tyrosine Kinases Retrospective cohort study medicine.disease Myasthenia gravis Regimen Neurology Tolerability Pediatrics Perinatology and Child Health Cohort Female Rituximab Neurology (clinical) business 030217 neurology & neurosurgery Follow-Up Studies medicine.drug |
| Zdroj: | Pediatric Neurology. 111:40-43 |
| ISSN: | 0887-8994 |
| Popis: | Background Juvenile myasthenia gravis is a pediatric autoimmune disorder of the neuromuscular junction associated with substantial morbidity, for which standard therapies are not always efficacious. The objective of our study was to assess the tolerability and efficacy of rituximab use in children with refractory juvenile myasthenia gravis. Methods We conduced a retrospective cohort study at a single tertiary care referral center to evaluate children with juvenile myasthenia gravis who were treated with rituximab. The clinical status of these participants before and after initiation of rituximab therapy was measured, focusing on numbers of hospital admissions, numbers of immunomodulatory or immunosuppressive medications needed, and Myasthenia Gravis Foundation of America severity class. Results Five children with juvenile myasthenia gravis were ascertained who received rituximab as part of their regimen, four of whom had elevated acetylcholine receptor antibodies and one of whom had elevated muscle-specific kinase antibodies. After initiation of rituximab therapy, all participants experienced reduced numbers of immunomodulatory medications during the follow-up period (mean 11.6 months). Four of the five subjects experienced fewer juvenile myasthenia gravis-related hospital admissions and reduced (improved) Myasthenia Gravis Foundation of America classes, with no subjects having moderate or severe symptoms following treatment with rituximab. No significant adverse events were recorded for any of the participants. Conclusion Rituximab was well-tolerated and efficacious in this juvenile myasthenia gravis cohort. The beneficial effect of rituximab was most pronounced in the one participant with muscle-specific kinase antibodies. |
| Databáze: | OpenAIRE |
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