Cargando…
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...
Autores principales: | , , , , , , , |
---|---|
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 |
_version_ | 1784606346777198592 |
---|---|
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. |
format | Online Article Text |
id | pubmed-8625142 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT auconimarina opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT papettilaura opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT ruscittoclaudia opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT ferillimichelaadanoris opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT ursittifabiana opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT sforzagiorgia opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT vigevanofederico opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge AT valerianimassimiliano opsoclonusmyoclonussyndromeinchildrenandadolescentsatherapeuticchallenge |