儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议

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儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议

中国研究型医院学会儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童神经内科专业委员会

文章导航 > 罕见病研究 > 2022 > 1(3): 304-310

中国研究型医院学会儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童神经内科专业委员会. 儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议[J]. 罕见病研究, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

引用本文:

中国研究型医院学会儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童神经内科专业委员会. 儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议[J]. 罕见病研究, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

Chinese Research Hospital Association- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Neurology Committee. Recommendations for Neuroblastoma Associated Opsoclonus-myoclonus-ataxia Syndrome in Childhood[J]. Journal of Rare Diseases, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

Citation:

Chinese Research Hospital Association- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Neurology Committee. Recommendations for Neuroblastoma Associated Opsoclonus-myoclonus-ataxia Syndrome in Childhood[J]. Journal of Rare Diseases, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

中国研究型医院学会儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童神经内科专业委员会. 儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议[J]. 罕见病研究, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

引用本文:

中国研究型医院学会儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童肿瘤专业委员会, 福棠儿童医学发展研究中心儿童神经内科专业委员会. 儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议[J]. 罕见病研究, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

Chinese Research Hospital Association- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Neurology Committee. Recommendations for Neuroblastoma Associated Opsoclonus-myoclonus-ataxia Syndrome in Childhood[J]. Journal of Rare Diseases, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

Citation:

Chinese Research Hospital Association- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Oncology Committee, FUTang Research Center of Pediatric Development- Children's Neurology Committee. Recommendations for Neuroblastoma Associated Opsoclonus-myoclonus-ataxia Syndrome in Childhood[J]. Journal of Rare Diseases, 2022, 1(3): 304-310. doi: 10.12376/j.issn.2097-0501.2022.03.012

儿童神经母细胞瘤相关性眼阵挛-肌阵挛-共济失调综合征的诊疗建议

doi: 10.12376/j.issn.2097-0501.2022.03.012

1.
国家儿童医学中心首都医科大学附属北京儿童医院儿童肿瘤中心肿瘤内科，北京 100045
2.
国家儿童医学中心首都医科大学附属北京儿童医院神经内科，北京 100045
3.
国家儿童医学中心首都医科大学附属北京儿童医院北京市儿童血液肿瘤重点实验室，北京 100045
4.
国家儿童医学中心首都医科大学附属北京儿童医院儿科重大疾病研究教育部重点实验室，北京 100045

通信作者：马晓莉^1，3，4，E-mail: mxl1123@vip.sina.com
方方²，E-mail: 13910150389@163.com

中图分类号: R729;R741
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出版历程
- 收稿日期: 2022-05-29
- 录用日期: 2022-06-06
- 网络出版日期: 2022-09-06

Recommendations for Neuroblastoma Associated Opsoclonus-myoclonus-ataxia Syndrome in Childhood

1.
Medical Oncology Department, Pediatric Oncology Center, National Center for Children's Health, Beijing Children's Hospital, Capital Merdical University, Beijing 100045, China
2.
Department of Neurology, National Center for Children's Health, Beijing Children's Hospital, Capital Merdical University, Beijing 100045, China
3.
Beijing Key Laboratory of Pediatric Hematology Ocology, National Center for Children's Health, Beijing Children's Hospital, Capital Merdical University, Beijing 100045, China
4.
Key Laboratory of Major Diseases in Children, Ministry of Education, National Center for Children's Health, Beijing Children's Hospital, Capital Merdical University, Beijing 100045, China

Corresponding authors: MA Xiaoli^{1, 3, 4}, E-mail: mxl1123@vip.sina.com
FANG Fang², E-mail: 13910150389@163.com

摘要: 眼阵挛-肌阵挛-共济失调综合征(OMAS)是一种罕见的神经系统疾病。该病常与神经母细胞瘤(NB)相关，我国儿童OMAS和NB分别由神经内科和肿瘤内、外科单独进行诊治。本组患儿中，NB通常预后良好，而OMAS易留有后遗症，但由于缺乏规范的诊治及随访，不利于患者疾病的控制。本文由多学科领域专家共同制订儿童NB相关性OMAS诊疗建议，从疾病的诊断、治疗、随访等方面进行阐述，希望通过对本组患儿的规范管理，改善患儿的预后。
- 眼阵挛-肌阵挛-共济失调综合征 /
- 神经母细胞瘤 /
- 诊断 /
- 治疗
Abstract: Opsoclonus-myoclonus-ataxia syndrome(OMAS) is a rare neurological disorder. This disease is often associated with neuroblastoma(NB). OMAS and neuroblastoma in children in China are treated separately by neurology, medical and surgical oncology. In this group of children, NB usually has a good prognosis, while OMAS is prone to sequelae, but the lack of standardized evaluation and follow-up is not conducive to the diagnosis and treatment of the disease. In this study, experts from multidisciplinary fields worked together to develop recommendations for the diagnosis, treatment and follow-up of NB associated OMAS in children, hoping to improve the prognosis of children through standardized management of this group of children.
- opsoclonus-myoclonus-ataxia syndrome /
- neuroblastoma /
- diagnosis /
- treatment
注释:

利益冲突：所有参与人员均声明不存在利益冲突。

表 1 眼阵挛-肌阵挛-共济失调综合征严重程度评分

Table 1. Opsoclonus-Myoclonus-Ataxia Syndrome severity scale

	功能	评分(分)
立位姿势
	与年龄相符的正常的站、坐平衡	0
	结合其年龄而言，轻微的站立不稳，站立时轻微的宽基底	1
	不给予支撑时无法站立，但没有支撑能独坐	2
	不使用手支撑或其他支撑就不能独坐	3
步态
	与年龄相符的正常行走	0
	结合其年龄而言，轻微的宽基底步态，但可在室内外独立行走	1
	只有或主要依靠他人或辅助设备支持时才能行走	2
	即使在他人或辅助设备的支持下也无法行走	3
手、臂功能
	正常且与年龄相符	0
	轻微的偶尔震颤或抽搐，无功能损害	1
	精细运动(比如小物件的夹取，使用铅笔等)持久损害，但是不太精确的操作任务(如玩更大的玩具、进食、穿衣等)正常或接近正常	2
	与其年龄相适应的操作任务存在明显困难	3
眼球阵挛
	无	0
	很少或仅在注视目标改变的时候出现	1
	频繁；频繁干扰注视和/或追视	2
	持续；持续地干扰注视及追视	3
情绪/行为
	正常	0
	轻度易怒，但可安抚好和/或轻度睡眠障碍，但容易解决	1
	易怒和睡眠障碍，严重干扰儿童和家庭的生活	2
	处于持续严重的情绪障碍	3
语言能力
	同同龄人，无下降	0
	不太清楚，语言发育停滞	1
	丧失一些词汇或语法结构(即从句子到短语)的表达能力，但仍能进行口头交流	2
	严重丧失口头交流和语言能力	3
对于18个月或以下的患儿，在评分时需考虑到下列能力：躯干垂直时能否保持头部始终直立；能否用每只手触摸和抓住物体；能否从俯卧位翻身到仰卧位并从仰卧位翻身到俯卧位

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参考文献(25)

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