Neuroblastoma With Opsoclonus-Myoclonus-Ataxia Syndrome: Role of Chemotherapy in the Management: Experience From a Tertiary Care Center in a Resource-limited Setting

Abstract

Children with neuroblastoma (NB) and opsoclonus-myoclonus-ataxia syndrome (OMAS) have a favorable oncologic outcome and overall survival. In contrast, despite intensive multidrug immunomodulation, the neurologic outcome is complicated by the relapsing nature of the neurologic symptoms and long-term neurobehavioral sequelae. Being associated with low-risk NB, there exists an ambiguity in the current literature regarding the administration of chemotherapy in these children. We reviewed our archives for children with NB-OMAS over a 22-year (January 1996 to January 2018) period. Eighteen children (10 female) with a median age at diagnosis of 23 months had NB-OMAS and were included. They had stage 1 (9/18; 50%), 2 (1/18; 5.5%), 3 (7/18; 39%), and 4 (1/18; 5.5%) disease according to the International Neuroblastoma Staging System. Multimodality therapy included surgery (16/18; 89%), chemotherapy (11/18; 61%), and immunomodulatory therapy (10/18; 55%). Complete oncologic remission was achieved in all children. Relapse of OMAS and presence of neurologic sequelae were observed in 1 (5.5%) and 5 (28%) cases, respectively. Presence of neurologic sequelae was significantly associated with low-tumor stage (P=0.036) and treatment without chemotherapy (P=0.003). Chemotherapy administration was the only variable significantly predicting a favorable neurologic outcome (95% confidence interval: 0.26-1.40, P=0.01). To conclude, our study including a limited cohort of patients highlights a favorable neurologic outcome associated with chemotherapy administration in children with NB-OMAS. However, further studies with larger sample size need to be conducted before drawing any definite conclusions.

Five Years Follow-up of Opsoclonus–Myoclonus–Ataxia Syndrome-Associated Neurogenic Tumors in Children

“…Speech and behavioral problems are especially detected. [19][20][21][22] The rate of neurological sequelae, including the persistence of OMAS symptoms, was reported as 56.5% in one study, while in another, it was reported between 20 and 60%. 13,22 In our study, the rate of sequelae is 33%.…”

“…However, as seen in the literature, the administration of chemotherapy is beneficial in selected cases in terms of intensifying possible immunosuppression and preventing long-term sequelae. 8,18,19 In our center, an institutional chemotherapy scheme consisting of six cycles of cisplatin, cyclophosphamide, and vincristine was applied to patients who could undergo subtotal tumor excision or were diagnosed with tru-cut biopsy or could not be obtained a definitive MYCN result (►Table 2). We consider that tumor or OMAS recurrences will be less after chemotherapy in such cases, and further studies are needed on this subject.…”

Profile and Outcome of Children with Opsoclonus Myoclonus Ataxia: A Tertiary Care Hospital Experience from India

“…16 17 Interestingly, Anand et al found chemotherapy administration (cyclophosphamide being a part of the regime) as the only variable significantly predicting a favorable neurologic outcome in neuroblastoma-OMAS. 14…”

“…Early diagnosis, prompt removal of underlying tumor, aggressive immunomodulation, and regular follow-up for relapses have been found to reduce morbidity. 6 7 8 9 10 14 However, delayed diagnosis and neurobehavioral sequalae are common. 5 6 7 10 Misdiagnosis as cerebellar ataxia, delayed detection, and removal of neurogenic tumors and immunomodulatory treatments have all been reported to affect outcomes.…”

“…5 6 7 10 Misdiagnosis as cerebellar ataxia, delayed detection, and removal of neurogenic tumors and immunomodulatory treatments have all been reported to affect outcomes. 6 7 10 14 Herein, we highlight these challenges and our experience in diagnosis and treatment of OMAS in a low- and middle-income country. We report the clinical profile, utility of investigations, and therapeutic outcome of children with OMAS consecutively recruited at a single tertiary care hospital from 2011 to 2021.…”