Successful use of tacrolimus for treatment-refractory neuroblastoma-associated opsoclonus-myoclonus-ataxia syndrome: A case series.

Autor: Delap S; Department of Hospital Pediatrics, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, Ohio State University College of Medicine, Columbus, Ohio, USA., Shah N; Department of Pediatrics, Ohio State University College of Medicine, Columbus, Ohio, USA.; Division of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, Ohio, USA., Kuns O; Center for Biobehavioral Health, Nationwide Children's Hospital, Columbus, Ohio, USA., Franklin B; Center for Biobehavioral Health, Nationwide Children's Hospital, Columbus, Ohio, USA., Skeens MA; Department of Pediatrics, Ohio State University College of Medicine, Columbus, Ohio, USA.; Division of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, Ohio, USA.; Center for Biobehavioral Health, Nationwide Children's Hospital, Columbus, Ohio, USA.
Jazyk: angličtina
Zdroj: Pediatric blood & cancer [Pediatr Blood Cancer] 2024 Apr; Vol. 71 (4), pp. e30903. Date of Electronic Publication: 2024 Feb 06.
DOI: 10.1002/pbc.30903
Abstrakt: 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.
(© 2024 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)
Databáze: MEDLINE
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