Anand Sachit, Agarwala Sandeep, Jain Vishesh, Bakhshi Sameer, Dhua Anjan, Gulati Sheffali, Seth Rachna, Srinivas Maddur, Jana Manisha, Kandasamy Devasenathipathy, Bhatnagar Veereshwar
Departments of Pediatric Surgery.
Department of Medical Oncology, BRAIRCH, AIIMS, New Delhi, India.
J Pediatr Hematol Oncol. 2021 Oct 1;43(7):e924-e929. doi: 10.1097/MPH.0000000000002131.
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.
患有神经母细胞瘤（NB）和眼阵挛-肌阵挛-共济失调综合征（OMAS）的儿童具有良好的肿瘤学预后和总体生存率。相比之下，尽管进行了强化多药免疫调节，但神经学预后因神经症状的复发性和长期神经行为后遗症而变得复杂。由于与低风险NB相关，目前文献中对于这些儿童化疗的应用存在模糊之处。我们回顾了1996年1月至2018年1月这22年间我们档案中患有NB-OMAS的儿童情况。18名儿童（10名女性）被纳入研究，诊断时的中位年龄为23个月，均患有NB-OMAS。根据国际神经母细胞瘤分期系统，他们的疾病分期为1期（9/18；50%）、2期（1/18；5.5%）、3期（7/18；39%）和4期（1/18；5.5%）。多模式治疗包括手术（16/18；89%）、化疗（11/18；61%）和免疫调节治疗（10/18；55%）。所有儿童均实现了完全肿瘤缓解。分别有1例（5.5%）和5例（28%）观察到OMAS复发和神经后遗症。神经后遗症的存在与低肿瘤分期（P=0.036）和未进行化疗的治疗（P=0.003）显著相关。化疗的应用是唯一显著预测良好神经学预后的变量（95%置信区间：0.26 - 1.40，P=0.01）。总之，我们纳入有限患者队列的研究强调了在患有NB-OMAS的儿童中，化疗应用与良好的神经学预后相关。然而，在得出任何明确结论之前，需要进行更大样本量的进一步研究。
