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:	Marina Auconi, Laura Papetti, Claudia Ruscitto, Michela Ada Noris Ferilli, Fabiana Ursitti, Giorgia Sforza, Federico Vigevano, Massimiliano Valeriani
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	opsoclonus-myoclonus syndrome pediatric neuroimmunological disorder neuroblastic tumors treatment outcome children Pediatrics RJ1-570
Acceso en línea:	https://doaj.org/article/a339353d51e94e9a8289d93d2cec185c
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spelling	oai:doaj.org-article:a339353d51e94e9a8289d93d2cec185c2021-11-25T17:13:59ZOpsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge10.3390/children81109652227-9067https://doaj.org/article/a339353d51e94e9a8289d93d2cec185c2021-10-01T00:00:00Zhttps://www.mdpi.com/2227-9067/8/11/965https://doaj.org/toc/2227-9067Opsoclonus-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 (<i>n</i> = 11). However, ACTH (<i>n</i> = 10), rituximab (<i>n</i> = 7), immunoglobulins (<i>n</i> = 4), cyclophosphamide (<i>n</i> = 3), and mycophenolate (<i>n</i> = 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.Marina AuconiLaura PapettiClaudia RuscittoMichela Ada Noris FerilliFabiana UrsittiGiorgia SforzaFederico VigevanoMassimiliano ValerianiMDPI AGarticleopsoclonus-myoclonus syndromepediatric neuroimmunological disorderneuroblastic tumorstreatmentoutcomechildrenPediatricsRJ1-570ENChildren, Vol 8, Iss 965, p 965 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	opsoclonus-myoclonus syndrome pediatric neuroimmunological disorder neuroblastic tumors treatment outcome children Pediatrics RJ1-570
spellingShingle	opsoclonus-myoclonus syndrome pediatric neuroimmunological disorder neuroblastic tumors treatment outcome children Pediatrics RJ1-570 Marina Auconi Laura Papetti Claudia Ruscitto Michela Ada Noris Ferilli Fabiana Ursitti Giorgia Sforza Federico Vigevano Massimiliano Valeriani Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
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 (<i>n</i> = 11). However, ACTH (<i>n</i> = 10), rituximab (<i>n</i> = 7), immunoglobulins (<i>n</i> = 4), cyclophosphamide (<i>n</i> = 3), and mycophenolate (<i>n</i> = 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.
format	article
author	Marina Auconi Laura Papetti Claudia Ruscitto Michela Ada Noris Ferilli Fabiana Ursitti Giorgia Sforza Federico Vigevano Massimiliano Valeriani
author_facet	Marina Auconi Laura Papetti Claudia Ruscitto Michela Ada Noris Ferilli Fabiana Ursitti Giorgia Sforza Federico Vigevano Massimiliano Valeriani
author_sort	Marina Auconi
title	Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge
title_short	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_sort	opsoclonus-myoclonus syndrome in children and adolescents: a therapeutic challenge
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/a339353d51e94e9a8289d93d2cec185c
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Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic Challenge

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