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Opsoclonus-myoclonus syndrome: clinical characteristics, therapeutic considerations, and prognostic factors in a Spanish paediatric cohort.
Neurologia. 2020 Jul 08 [Online ahead of print]N

Abstract

INTRODUCTION

Opsoclonus-myoclonus-ataxia syndrome is a rare neuroinflammatory disorder with onset during childhood; aetiology may be paraneoplastic, para-infectious, or idiopathic. No biomarkers have yet been identified, and diagnosis is clinical. Better cognitive prognosis appears to be related to early onset of immunomodulatory therapy.

METHODS

We describe the epidemiological, clinical, therapeutic, and long-term prognostic characteristics of a cohort of 20 Spanish patients.

RESULTS

The mean age of onset was 21 months (range, 2-59). Ataxia and opsoclonus were the most frequent symptoms both at disease onset and throughout disease progression. The mean time from onset to diagnosis was 1.1 months. Neuroblast lineage tumours were detected in 45% of patients; these were treated with surgical resection in 7 cases and chemotherapy in 2. Cerebrospinal fluid analysis revealed pleocytosis in 4 cases (25%) and neither antineuronal antibodies nor oligoclonal bands were detected in any patient. Immunomodulatory drugs were used in all cases. Nine patients started combined immunomodulatory treatment at the time of diagnosis, and 5 patients after a mean of 2.2 months. In the long term, 6 of the 10 patients followed up for more than 5 years presented mild or moderate cognitive sequelae. Four patients presented relapses, generally coinciding with the decrease of corticosteroid doses.

CONCLUSIONS

Early initiation of immunotherapy, as well as triple combination therapy, where needed, was associated with a lower frequency of cognitive impairment 2 years after onset.

Authors+Show Affiliations

Sección de Neuropediatría. Servicio de Pediatría. Hospital Infantil Universitario Niño Jesús, Madrid, España; Grupo Clínico vinculado (GCV14/ER/6) al Centro de Investigación Biomédica en Red para Enfermedades Raras (CIBERER). Instituto de Salud Carlos III, Madrid, España. Electronic address: veronica.cantarin@salud.madrid.org.Sección de Neuropediatría. Servicio de Pediatría. Hospital Infantil Universitario Niño Jesús, Madrid, España.Unidad de Neuropediatría. Servicio de Pediatría. Complejo Hospitalario de Navarra. Servicio Navarro de Salud, Pamplona, España; Navarrabiomed-Fundación Miguel Servet, Pamplona, España.Unidad de Neuropediatría. Servicio de Pediatría. Hospital Universitario Río Hortega, Valladolid, España.Unidad de Neuropediatría. Servicio de Pediatría. Hospital Universitario Virgen de las Nieves, Granada, España.Unidad de Neuropediatría. Servicio de Pediatría. Hospital Universitario Araba. Servicio Vasco de Salud, Vitoria-Gasteiz, España.Unidad de Neuropediatría. Servicio de Pediatría. Hospital Universitario Donostia. Servicio Vasco de Salud, Donosti, España.Unidad de Neuropediatría. Servicio de Pediatría. Complejo Hospitalario de Navarra. Servicio Navarro de Salud, Pamplona, España; Navarrabiomed-Fundación Miguel Servet, Pamplona, España.Unidad de Oncología Infantil. Servicio de Pediatría. Complejo Hospitalario de Navarra. Servicio Navarro de Salud, Pamplona, España.Sección de Neuropediatría. Servicio de Pediatría. Hospital Infantil Universitario Niño Jesús, Madrid, España; Grupo Clínico vinculado (GCV14/ER/6) al Centro de Investigación Biomédica en Red para Enfermedades Raras (CIBERER). Instituto de Salud Carlos III, Madrid, España.

Pub Type(s)

Journal Article

Language

eng spa

PubMed ID

32653103

Citation

Cantarín-Extremera, V, et al. "Opsoclonus-myoclonus Syndrome: Clinical Characteristics, Therapeutic Considerations, and Prognostic Factors in a Spanish Paediatric Cohort." Neurologia (Barcelona, Spain), 2020.
Cantarín-Extremera V, Jiménez-Legido M, Aguilera-Albesa S, et al. Opsoclonus-myoclonus syndrome: clinical characteristics, therapeutic considerations, and prognostic factors in a Spanish paediatric cohort. Neurologia. 2020.
Cantarín-Extremera, V., Jiménez-Legido, M., Aguilera-Albesa, S., Hedrera-Fernández, A., Arrabal-Fernández, L., Gorría-Redondo, N., Martí-Carrera, I., Yoldi-Pedtri, M. E., Sagaseta-De Ilúrdoz, M., & González-Gutiérrez-Solana, L. (2020). Opsoclonus-myoclonus syndrome: clinical characteristics, therapeutic considerations, and prognostic factors in a Spanish paediatric cohort. Neurologia (Barcelona, Spain). https://doi.org/10.1016/j.nrl.2020.04.025
Cantarín-Extremera V, et al. Opsoclonus-myoclonus Syndrome: Clinical Characteristics, Therapeutic Considerations, and Prognostic Factors in a Spanish Paediatric Cohort. Neurologia. 2020 Jul 8; PubMed PMID: 32653103.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Opsoclonus-myoclonus syndrome: clinical characteristics, therapeutic considerations, and prognostic factors in a Spanish paediatric cohort. AU - Cantarín-Extremera,V, AU - Jiménez-Legido,M, AU - Aguilera-Albesa,S, AU - Hedrera-Fernández,A, AU - Arrabal-Fernández,L, AU - Gorría-Redondo,N, AU - Martí-Carrera,I, AU - Yoldi-Pedtri,M E, AU - Sagaseta-De Ilúrdoz,M, AU - González-Gutiérrez-Solana,L, Y1 - 2020/07/08/ PY - 2019/09/24/received PY - 2020/03/05/revised PY - 2020/04/01/accepted PY - 2020/7/13/entrez PY - 2020/7/13/pubmed PY - 2020/7/13/medline KW - Immunotherapy KW - Inmunoterapia KW - MIBG KW - Neuroblastoma KW - Neurocognitive KW - Neurocognitivo KW - Neuroinflamación KW - Neuroinflammation KW - Opsoclonus-myoclonus-ataxia syndrome KW - Síndrome opsoclono-mioclono-ataxia JF - Neurologia (Barcelona, Spain) JO - Neurologia N2 - INTRODUCTION: Opsoclonus-myoclonus-ataxia syndrome is a rare neuroinflammatory disorder with onset during childhood; aetiology may be paraneoplastic, para-infectious, or idiopathic. No biomarkers have yet been identified, and diagnosis is clinical. Better cognitive prognosis appears to be related to early onset of immunomodulatory therapy. METHODS: We describe the epidemiological, clinical, therapeutic, and long-term prognostic characteristics of a cohort of 20 Spanish patients. RESULTS: The mean age of onset was 21 months (range, 2-59). Ataxia and opsoclonus were the most frequent symptoms both at disease onset and throughout disease progression. The mean time from onset to diagnosis was 1.1 months. Neuroblast lineage tumours were detected in 45% of patients; these were treated with surgical resection in 7 cases and chemotherapy in 2. Cerebrospinal fluid analysis revealed pleocytosis in 4 cases (25%) and neither antineuronal antibodies nor oligoclonal bands were detected in any patient. Immunomodulatory drugs were used in all cases. Nine patients started combined immunomodulatory treatment at the time of diagnosis, and 5 patients after a mean of 2.2 months. In the long term, 6 of the 10 patients followed up for more than 5 years presented mild or moderate cognitive sequelae. Four patients presented relapses, generally coinciding with the decrease of corticosteroid doses. CONCLUSIONS: Early initiation of immunotherapy, as well as triple combination therapy, where needed, was associated with a lower frequency of cognitive impairment 2 years after onset. SN - 1578-1968 UR - https://www.unboundmedicine.com/medline/citation/32653103/Opsoclonus-myoclonus_syndrome:_clinical_characteristics,_therapeutic_considerations,_and_prognostic_factors_in_a_Spanish_paediatric_cohort L2 - http://www.elsevier.es/en/linksolver/ft/pii/S0213-4853(20)30137-7 DB - PRIME DP - Unbound Medicine ER -