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Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge

Opsoclonus-myoclonus syndrome (OMS) is a neurological non-fatal disease that usually responds to immunotherapies. However, the real challenge is to counteract the high frequency of relapses and long-term developmental sequelae. Since the OMS is extremely rare, a common consensus regarding therapeuti...

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Autores principales: Auconi, Marina, Papetti, Laura, Ruscitto, Claudia, Ferilli, Michela Ada Noris, Ursitti, Fabiana, Sforza, Giorgia, Vigevano, Federico, Valeriani, Massimiliano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8625142/
https://www.ncbi.nlm.nih.gov/pubmed/34828678
http://dx.doi.org/10.3390/children8110965
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author Auconi, Marina
Papetti, Laura
Ruscitto, Claudia
Ferilli, Michela Ada Noris
Ursitti, Fabiana
Sforza, Giorgia
Vigevano, Federico
Valeriani, Massimiliano
author_facet Auconi, Marina
Papetti, Laura
Ruscitto, Claudia
Ferilli, Michela Ada Noris
Ursitti, Fabiana
Sforza, Giorgia
Vigevano, Federico
Valeriani, Massimiliano
author_sort Auconi, Marina
collection PubMed
description Opsoclonus-myoclonus syndrome (OMS) is a neurological non-fatal disease that usually responds to immunotherapies. However, the real challenge is to counteract the high frequency of relapses and long-term developmental sequelae. Since the OMS is extremely rare, a common consensus regarding therapeutic guidelines is still lacking. The goals of this study were to test whether ACTH was superior to other immunotherapies and to investigate whether an early treatment could improve the outcome. Sixteen children affected by OMS were retrospectively reviewed. Eight children had a neuroblastic tumor. The other eight patients were affected by non-paraneoplastic OMS. Overall, the most commonly used treatment was corticotherapy (n = 11). However, ACTH (n = 10), rituximab (n = 7), immunoglobulins (n = 4), cyclophosphamide (n = 3), and mycophenolate (n = 2) were also administered. ACTH was associated with a high percentage of patients who healed (80%) and, as a first-line therapy, was associated with a lower incidence of relapses. An early treatment was associated with a favorable long-term outcome. Long-term sequelae occurred in 42% of patients who were treated early and in all of those who were treated late. It is advisable for the affected children to be identified at an early time, as they may benefit from an early treatment. ACTH represents an effective treatment with a high probability of recovery and low rate of relapses.
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spelling pubmed-86251422021-11-27 Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge Auconi, Marina Papetti, Laura Ruscitto, Claudia Ferilli, Michela Ada Noris Ursitti, Fabiana Sforza, Giorgia Vigevano, Federico Valeriani, Massimiliano Children (Basel) Case Report Opsoclonus-myoclonus syndrome (OMS) is a neurological non-fatal disease that usually responds to immunotherapies. However, the real challenge is to counteract the high frequency of relapses and long-term developmental sequelae. Since the OMS is extremely rare, a common consensus regarding therapeutic guidelines is still lacking. The goals of this study were to test whether ACTH was superior to other immunotherapies and to investigate whether an early treatment could improve the outcome. Sixteen children affected by OMS were retrospectively reviewed. Eight children had a neuroblastic tumor. The other eight patients were affected by non-paraneoplastic OMS. Overall, the most commonly used treatment was corticotherapy (n = 11). However, ACTH (n = 10), rituximab (n = 7), immunoglobulins (n = 4), cyclophosphamide (n = 3), and mycophenolate (n = 2) were also administered. ACTH was associated with a high percentage of patients who healed (80%) and, as a first-line therapy, was associated with a lower incidence of relapses. An early treatment was associated with a favorable long-term outcome. Long-term sequelae occurred in 42% of patients who were treated early and in all of those who were treated late. It is advisable for the affected children to be identified at an early time, as they may benefit from an early treatment. ACTH represents an effective treatment with a high probability of recovery and low rate of relapses. MDPI 2021-10-26 /pmc/articles/PMC8625142/ /pubmed/34828678 http://dx.doi.org/10.3390/children8110965 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Case Report
Auconi, Marina
Papetti, Laura
Ruscitto, Claudia
Ferilli, Michela Ada Noris
Ursitti, Fabiana
Sforza, Giorgia
Vigevano, Federico
Valeriani, Massimiliano
Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_full Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_fullStr Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_full_unstemmed Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_short Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_sort opsoclonus-myoclonus syndrome in children and adolescents: a therapeutic challenge
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8625142/
https://www.ncbi.nlm.nih.gov/pubmed/34828678
http://dx.doi.org/10.3390/children8110965
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