Successful use of tacrolimus for treatment-refractory neuroblastoma-associated opsoclonus-myoclonus-ataxia syndrome: A case series.
Pediatr Blood Cancer
; 71(4): e30903, 2024 Apr.
Article
em En
| MEDLINE
| ID: mdl-38321586
ABSTRACT
Opsoclonus-myoclonus-ataxia syndrome (OMAS) is an autoimmune central nervous system disorder, primarily manifesting as a paraneoplastic sequalae to neuroblastoma, and characterized by motor disorders and behavioral disturbances. OMAS is typified by aberrant B-cell and T-cell activation. Current treatment involves immunosuppression using corticosteroids, intravenous immunoglobulin, and rituximab. However, these approaches often lead to treatment-related toxicities and symptomatic recurrences with chronic neurocognitive impairment. We treated three children with refractory neuroblastoma-associated OMAS with tacrolimus, a T-cell-targeting calcineurin inhibitor, effectively controlling symptoms within a month and enabling the discontinuation of immunosuppression with minimal side effects. Tacrolimus shows promise as a therapeutic option for refractory OMAS.
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Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Transtornos da Motilidade Ocular
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Síndrome de Opsoclonia-Mioclonia
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Neuroblastoma
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article