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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| Format: | Article |
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MDPI AG
2021-10-01
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| Series: | Children |
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| Online Access: | https://www.mdpi.com/2227-9067/8/11/965 |
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| 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 |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-03-10T05:36:33Z |
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| id | doaj.art-a339353d51e94e9a8289d93d2cec185c |
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| issn | 2227-9067 |
| language | English |
| last_indexed | 2024-03-10T05:36:33Z |
| publishDate | 2021-10-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Children |
| spelling | doaj.art-a339353d51e94e9a8289d93d2cec185c2023-11-22T22:54:33ZengMDPI AGChildren2227-90672021-10-0181196510.3390/children8110965Opsoclonus-Myoclonus Syndrome in Children and Adolescents: A Therapeutic ChallengeMarina Auconi0Laura Papetti1Claudia Ruscitto2Michela Ada Noris Ferilli3Fabiana Ursitti4Giorgia Sforza5Federico Vigevano6Massimiliano Valeriani7Child Neurology and Psychiatry Unit, Systems Medicine Department, Tor Vergata University of Rome, 00133 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyChild Neurology and Psychiatry Unit, Systems Medicine Department, Tor Vergata University of Rome, 00133 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyChild Neurology Unit, Neuroscience Department, Bambino Gesù Children’s Hospital, IRCCS, 00165 Rome, ItalyOpsoclonus-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.https://www.mdpi.com/2227-9067/8/11/965opsoclonus-myoclonus syndromepediatric neuroimmunological disorderneuroblastic tumorstreatmentoutcomechildren |
| spellingShingle | 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 Children opsoclonus-myoclonus syndrome pediatric neuroimmunological disorder neuroblastic tumors treatment outcome children |
| 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 | opsoclonus-myoclonus syndrome pediatric neuroimmunological disorder neuroblastic tumors treatment outcome children |
| url | https://www.mdpi.com/2227-9067/8/11/965 |
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