52例颅内非典型畸胎样/横纹肌样瘤的临床特征及生存分析

doi:10.3969/j.issn.1674-8115.2021.09.013

上海交通大学学报(医学版) ›› 2021, Vol. 41 ›› Issue (9): 1222-1227.doi: 10.3969/j.issn.1674-8115.2021.09.013

• 论著 · 临床研究 • 上一篇

52例颅内非典型畸胎样/横纹肌样瘤的临床特征及生存分析

徐艳丽¹(), 管文斌², 茅伟伟³, 朱传营⁴, 张勤¹, 袁晓军¹, 谈珍¹()

^1.上海交通大学医学院附属新华医院儿血液肿瘤科，上海 200092
^2.上海交通大学医学院附属新华医院病理科，上海 200092
^3.上海交通大学医学院附属新华医院儿神经外科，上海 200092
^4.上海交通大学医学院附属新华医院肿瘤科，上海 200092

收稿日期:2020-11-03 出版日期:2021-08-09 发布日期:2021-08-09
通讯作者: 谈珍 E-mail:xhxexy@126.com;tanz2008@126.com
作者简介:徐艳丽（1987—），女，住院医师，硕士；电子信箱：xhxexy@126.com。
基金资助:
上海市高级中西医结合人才培养计划［ZY（2018-2020）-RCPY-2018］;上海交通大学医学院附属新华医院临床研究培育基金(17CSY04)

Clinical characteristics and survival analysis of 52 cases with intracranial atypical teratoid/rhabdoid tumor

Yan-li XU¹(), Wen-bin GUAN², Wei-wei MAO³, Chuan-ying ZHU⁴, Qin ZHANG¹, Xiao-jun YUAN¹, Zhen TAN¹()

^1.Department of Pediatric Hematology/Oncology, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China
^2.Department of Pathology, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China
^3.Department of Pediatric Neurosurgery, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China
^4.Department of Oncology, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China

Received:2020-11-03 Online:2021-08-09 Published:2021-08-09
Contact: Zhen TAN E-mail:xhxexy@126.com;tanz2008@126.com
Supported by:
Senior Talents Training Plan of Shanghai Advanced Traditional Chinese Medicine and Western Medicine Combination [ZY(2018-2020)-RCPY-2018];Clinical Research Cultivation Fund of Xinhua Hospital, Shanghai Jiao Tong University School of Medicine(17CSY04)

摘要/Abstract

摘要：

目的·总结近8年诊治过的颅内非典型畸胎样/横纹肌样瘤（atypical teratoid/rhabdoid tumor，AT/RT）患儿的临床特征及生存情况，并分析其生存相关因素，为进一步分层治疗提供依据。方法·选择2012年10月—2020年1月在上海交通大学医学院附属新华医院救治的AT/RT患儿52例，记录其临床资料及随访情况。采用Kaplan-Meier法分析患儿3年无进展生存率（progression-free survival，PFS）和3年总体生存率（overall survival，OS）。根据患儿年龄，将其分为≥3岁组和<3岁组，比较2组患儿的临床特征及生存情况。采用单因素及多因素Cox比例风险回归模型分析临床特征对患儿总体生存的影响。根据不同的生存结局，探讨患儿免疫组织化学指标表达的意义。结果·AT/RT患儿中，男性34例、女性18例，65.4%的患儿小于3岁，平均发病年龄为（35.2±28.2）个月，中位生存时间为7.7个月。患儿的3年PFS及3年OS分别为（29.3±7.6）%和（30.4±7.6）%。2组患儿在接受放射治疗（放疗）及化学治疗（化疗）、出现严重并发症、3年PFS和3年OS间差异具有统计学意义（均P<0.05）。单因素Cox回归分析显示，年龄<3岁、原发肿瘤侵犯椎管、有播散转移、次全切除肿瘤、未接受放疗或化疗的患儿的生存风险较大（均P<0.05）。多因素Cox回归分析提示，肿瘤有播散转移是患儿总体生存的独立危险因素（P=0.024）。对患儿的免疫组织化学指标进行统计，结果显示存活患儿的SYN阳性表达明显高于死亡患儿（P=0.003）。结论·年龄小于3岁的AT/RT患儿存在较多严重并发症且总体生存率较低，需要施行更精细的分层治疗方式及多样化的治疗手段。

关键词: 非典型畸胎样/横纹肌样瘤, 恶性脑肿瘤, 生存分析

Abstract:

Objective·To summarize the clinical characteristics and survival status of children with intracranial atypical teratoid/rhabdoid tumor (AT/RT) diagnosed and treated in recent 8 years, and analyze the survival-related factors, so as to provide basis for further therapeutic stratification.

Methods·From October 2012 to January 2020, 52 AT/RT children treated in Xinhua Hospital, Shanghai Jiao Tong University School of Medicine were selected. Their clinical data and follow-up were recorded and their 3-year progression-free survival (PFS) and 3-year overall survival (OS) were analyzed by Kaplan-Meier method. According to the age of the children, they were divided into ≥3-year-old group and <3-year-old group. The clinical characteristics and survival status of the two groups were compared. Univariate and multivariate Cox proportional hazards regression models were used to analyze the impact of clinical characteristics on overall survival. The significance of immunohistochemical indicators in children with different survival outcomes was explored.

Results·There were 34 males and 18 females in AT/RT children, 65.4% of which were younger than 3 years old, and the average age of onset was (35.2±28.2) months. The median survival time was 7.7 months, and 3-year PFS and 3-year OS was (29.3±7.6)% and (30.4±7.6)%, respectively. There were significant differences between the two groups in receiving radiotherapy and chemotherapy, serious complications, 3-year PFS and 3-year OS (all P<0.05). Univariate Cox regression analysis showed that the survival risk of children, who was less than 3 years old and underwent primary tumor invading the spinal canal, tumor metastasis, and partial resection, without radiotherapy or chemotherapy, was increasing (all P<0.05). Multivariate Cox regression analysis showed that tumor metastasis was an independent risk factor for overall survival of children (P=0.024). The results from immunohistochemistry showed that the positive expression of SYN in survival children was significantly higher than that in dead children (P=0.003).

Conclusion·Children younger than 3 years old with AT/RT have more severe complications and lower overall survival rate, so it is necessary to implement more accurate therapeutic stratification and diversified treatment methods.

Key words: atypical teratoid/rhabdoid tumor (AT/RT), malignant brain tumor, survival analysis

中图分类号:

R739.41

徐艳丽, 管文斌, 茅伟伟, 朱传营, 张勤, 袁晓军, 谈珍. 52例颅内非典型畸胎样/横纹肌样瘤的临床特征及生存分析[J]. 上海交通大学学报(医学版), 2021, 41(9): 1222-1227.

Yan-li XU, Wen-bin GUAN, Wei-wei MAO, Chuan-ying ZHU, Qin ZHANG, Xiao-jun YUAN, Zhen TAN. Clinical characteristics and survival analysis of 52 cases with intracranial atypical teratoid/rhabdoid tumor[J]. JOURNAL OF SHANGHAI JIAOTONG UNIVERSITY (MEDICAL SCIENCE), 2021, 41(9): 1222-1227.

图/表 5

参考文献 17

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Item	<3-year-old group （n=34）	≥3-year-old group （n=18）	P value
Gender/n（%）			0.637
Male	23 （67.6）	11 （61.1）
Female	11 （32.4）	7 （38.9）
Primary site of tumor/n（%）			0.610
Supratentorial	17 （50.0）	8 （44.4）
Infratentorial	13 （38.2）	6 （33.3）
Spinal canal	4 （11.8）	4 （22.2）
Metastasis/n（%）			0.733
M0	27 （79.4）	15 （83.3）
M+	7 （20.6）	3 （16.7）
Treatment method/n（%）
Surgical excision	27 （79.4）	15 （83.3）	0.929
Radiotherapy	9 （26.5）	16 （88.9）	0.000
Chemotherapy	9 （26.5）	11 （61.1）	0.015
Severe complication/n（%）	7 （20.6）	0 （0）	0.038
Survival probability/%
3-year PFS	22.1±8.7	30.6±12.2	0.029
3-year OS	24.3±7.4	37.3±12.6	0.044

Item	<3-year-old group （n=34）	≥3-year-old group （n=18）	P value
Gender/n（%）			0.637
Male	23 （67.6）	11 （61.1）
Female	11 （32.4）	7 （38.9）
Primary site of tumor/n（%）			0.610
Supratentorial	17 （50.0）	8 （44.4）
Infratentorial	13 （38.2）	6 （33.3）
Spinal canal	4 （11.8）	4 （22.2）
Metastasis/n（%）			0.733
M0	27 （79.4）	15 （83.3）
M+	7 （20.6）	3 （16.7）
Treatment method/n（%）
Surgical excision	27 （79.4）	15 （83.3）	0.929
Radiotherapy	9 （26.5）	16 （88.9）	0.000
Chemotherapy	9 （26.5）	11 （61.1）	0.015
Severe complication/n（%）	7 （20.6）	0 （0）	0.038
Survival probability/%
3-year PFS	22.1±8.7	30.6±12.2	0.029
3-year OS	24.3±7.4	37.3±12.6	0.044

Item	Univariate analysis			Multivariate analysis
Item	HR	OR（95% CI）	P value	HR	OR（95% CI）	P value
Gender
Male （n=34）	Ref	‒	‒	‒	‒	‒
Female （n=18）	1.292	0.627‒2.660	0.487	‒	‒	‒
Age
<3 years （n=34）	2.266	1.055‒4.866	0.036	0.813	0.210‒3.144	0.765
≥3 years （n=18）	Ref	‒	‒	Ref	‒	‒
Primary site of tumor
Supratentorial （n=25）	Ref	‒	‒	Ref	‒	‒
Infratentorial （n=19）	1.315	0.599‒2.889	0.495	1.246	0.520‒2.983	0.622
Spinal canal （n=8）	2.735	1.104‒6.775	0.030	0.270	0.040‒1.833	0.180
Metastasis
M+ （n=42）	2.891	1.353‒6.178	0.006	3.772	1.191‒11.945	0.024
M0 （n=10）	Ref	‒	‒	Ref	‒	‒
Surgical resection
Complete excision （n=42）	Ref	‒	‒	Ref	‒	‒
Partial excision （n=10）	4.017	1.749‒9.228	0.001	5.336	0.814‒34.962	0.081
Radiotherapy
Yes （n=25）	Ref	‒	‒	Ref	‒	‒
No （n=27）	4.763	2.245‒10.105	0.000	3.185	0.758‒13.371	0.114
Chemotherapy
Yes （n=20）	Ref	‒	‒	Ref	‒	‒
No （n=32）	4.378	1.984‒9.659	0.000	2.752	0.958‒7.911	0.060

Item	Univariate analysis			Multivariate analysis
Item	HR	OR（95% CI）	P value	HR	OR（95% CI）	P value
Gender
Male （n=34）	Ref	‒	‒	‒	‒	‒
Female （n=18）	1.292	0.627‒2.660	0.487	‒	‒	‒
Age
<3 years （n=34）	2.266	1.055‒4.866	0.036	0.813	0.210‒3.144	0.765
≥3 years （n=18）	Ref	‒	‒	Ref	‒	‒
Primary site of tumor
Supratentorial （n=25）	Ref	‒	‒	Ref	‒	‒
Infratentorial （n=19）	1.315	0.599‒2.889	0.495	1.246	0.520‒2.983	0.622
Spinal canal （n=8）	2.735	1.104‒6.775	0.030	0.270	0.040‒1.833	0.180
Metastasis
M+ （n=42）	2.891	1.353‒6.178	0.006	3.772	1.191‒11.945	0.024
M0 （n=10）	Ref	‒	‒	Ref	‒	‒
Surgical resection
Complete excision （n=42）	Ref	‒	‒	Ref	‒	‒
Partial excision （n=10）	4.017	1.749‒9.228	0.001	5.336	0.814‒34.962	0.081
Radiotherapy
Yes （n=25）	Ref	‒	‒	Ref	‒	‒
No （n=27）	4.763	2.245‒10.105	0.000	3.185	0.758‒13.371	0.114
Chemotherapy
Yes （n=20）	Ref	‒	‒	Ref	‒	‒
No （n=32）	4.378	1.984‒9.659	0.000	2.752	0.958‒7.911	0.060

Item	Survival outcome		P value
Item	Survival （n=16）	Death （n=29）	P value
INI -1 deletion/n（%）	15 （93.8）	28 （96.6）	0.331
Ki67 positive expression/%	49.6±18.4	41.7±18.6	0.089
P53 abnormal expression/n（%）	2 （22.2）	7 （24.1）	0.470
GFAP positive expression/n（%）	11 （91.7）	22 （75.9）	0.110
VIM positive expression/n（%）	7 （100.0）	20 （100.0）	0.500
SMA positive expression/n（%）	0 （0）	3 （15.8）	0.274
EMA positive expression/n（%）	8 （61.5）	24 （82.8）	0.084
CD99 positive expression/n（%）	4 （44.4）	15 （71.4）	0.098
SYN positive expression/n（%）	8 （88.9）	10 （34.5）	0.003
OLIG2 positive expression/n（%）	4 （50.0）	13 （44.8）	0.404

52例颅内非典型畸胎样/横纹肌样瘤的临床特征及生存分析

Clinical characteristics and survival analysis of 52 cases with intracranial atypical teratoid/rhabdoid tumor

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