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Article(id=1199335052925567024, tenantId=1146029695717560320, journalId=1189873630562394117, issueId=1199335049175859209, articleNumber=null, orderNo=null, doi=10.11855/j.issn.0577-7402.2030.2023.0630, pmid=null, cstr=null, oa=null, hot=null, price=null, onlineType=0, articleFormat=0, articleType=null, articleTypeStr=research-article, receivedDate=1664294400000, receivedDateStr=2022-09-28, revisedDate=null, revisedDateStr=null, acceptedDate=1669478400000, acceptedDateStr=2022-11-27, onlineDate=1763873359185, onlineDateStr=2025-11-23, pubDate=1711555200000, pubDateStr=2024-03-28, doiRegisterDate=null, doiRegisterDateStr=null, onlineIssueDate=1763873359185, onlineIssueDateStr=2025-11-23, onlineJustAcceptDate=null, onlineJustAcceptDateStr=null, onlineFirstDate=null, onlineFirstDateStr=null, sourceXml=null, magXml=null, createTime=1763873359185, creator=13701087609, updateTime=1763873359185, updator=13701087609, issue=Issue{id=1199335049175859209, tenantId=1146029695717560320, journalId=1189873630562394117, year='2024', volume='49', issue='3', pageStart='245', pageEnd='366', issueExtLink='null', onlineDate='null', pubDate='null', beforeIssueId=null, nextIssueId=null, price=null, status=1, issueComplete=1, articleOrder=1, issueType=-1, specialIssue=0, createTime=1763873358291, creator=13701087609, updateTime=1763874044185, updator=13701087609, preIssue=null, nextIssue=null, ext={EN=IssueExt(id=1199337926086721596, tenantId=1146029695717560320, journalId=1189873630562394117, issueId=1199335049175859209, language=EN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=), CN=IssueExt(id=1199337926086721597, tenantId=1146029695717560320, journalId=1189873630562394117, issueId=1199335049175859209, language=CN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=)}, issueFiles=null}, startPage=302, endPage=307, ext={EN=ArticleExt(id=1199335053202391095, articleId=1199335052925567024, tenantId=1146029695717560320, journalId=1189873630562394117, language=EN, title=Efficacy evaluation and prognostic factors analysis of retinoblastoma based on propensity score inverse probability weighting method, columnId=1190310109000602400, journalTitle=Medical Journal of Chinese People’s Liberation Army, columnName=Clinical Research, runingTitle=null, highlight=null, articleAbstract=

Objective To evaluate the efficacy of surgery, chemotherapy and surgery combined chemotherapy for retinoblastoma (RB), and analyze the prognostic factors of RB patients. Methods Clinical data of 1188 RB patients registered in the Surveillance, Epidemiology and End Results (SEER) database from January 2000 to December 2019 were retrospectively analyzed. The baseline characteristics of patients treated with surgery, chemotherapy or surgery combined with chemotherapy were balanced by inverse probability of treatment weighting (IPTW). Log-rank test analysis was used to compare the survival probability of patients in the 3 groups, and Cox regression models were used to analyse the factors influencing the prognosis of RB patients. Results A total of 1188 RB cases were included in this study, including 426 cases in surgery group, 200 cases in chemotherapy group and 562 cases in surgery combined with chemotherapy group. After IPTW weighting, baseline data such as age, sex and race were balanced (P>0.05). Log-rank test results showed that the survival curves of the three groups were significantly different before and after weighting (P<0.05). After weighted, the survival of patients in surgery group was significantly better than that in chemotherapy group and surgery combined chemotherapy group (P<0.05), and there was no statistical significance between chemotherapy group and surgery combined chemotherapy group (P>0.05).The weighted patient survival probability at 1st, 3rd and 5th years were 99.7%, 98.9% and 98.6% in surgery group; 97.4%, 95.8% and 95.8% in chemotherapy group; and 97.9%, 95.8% and 95.0% in surgery combined chemotherapy group. Cox regression analysis showed that compared with surgery group, the specific risk ratio of death was 1.367 (95%CI 1.100-1.700) in chemotherapy group and 1.132 (95%CI 0.963-1.330) in combined chemotherapy group. Compared with patients with 1 RB lesion, the patient-specific mortality risk ratio for patients with 2 or more RB lesions was 0.399 (95%CI 0.268-0.594). Conclusions Patients with RB have higher survival rates probability after treatment. After controlling the influence of age, sex and other factors, the effect of surgery was better among the three treatment methods. Multifocality may be an independent prognostic factor in RB patients.

, correspAuthors=Zhi-Hong Wu, authorNote=null, correspAuthorsNote=
E-mail:
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目的 评估手术、化疗及手术联合化疗3种治疗方式对视网膜母细胞瘤(RB)的疗效，分析RB患者的预后影响因素。方法 选取2000年1月1日-2019年12月31日美国监测、流行病学和最终结果(SEER)数据库登记的RB患者1188例临床资料进行回顾性分析。采用倾向性评分逆概率加权法(IPTW)对接受手术、化疗或手术联合化疗治疗的3组患者基线特征进行平衡。采用Log-rank检验比较3组患者的生存概率，采用Cox回归模型分析RB患者预后影响因素。结果 共纳入RB 1188例，其中手术组426例，化疗组200例，手术联合化疗组562例。经IPTW加权后，患者的年龄、性别、种族等基线资料均达到平衡(P>0.05)。Log-rank检验结果显示，加权前后3组生存曲线比较差异均有统计学意义(P<0.05)；加权后，手术组患者生存情况明显优于化疗组和手术联合化疗组(P<0.05)，化疗组与手术联合化疗组比较差异无统计学意义(P>0.05)。加权后患者1年、3年和5年的生存概率为：手术组99.7%、98.9%和98.6%；化疗组97.4%、95.8%和95.8%；手术联合化疗组97.9%、95.8%和95.0%。Cox回归分析结果显示，与手术组比较，化疗组患者特异性死亡风险比为1.367(95%CI 1.100~1.700)，手术联合化疗组特异性死亡风险比为1.132(95%CI 0.963~1.330)；与RB病灶为1个的患者比较，RB病灶数≥2个的患者特异性死亡风险比为0.399(95%CI 0.268~0.594)。结论 RB患者接受治疗后生存概率较高；在控制年龄、性别等因素的影响后，3种治疗方式中手术的疗效更好；多灶性可能是RB患者预后的独立影响因素。

, correspAuthors=吴志鸿, authorNote=null, correspAuthorsNote=
吴志鸿，E-mail：
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石丽娟，硕士研究生，主要从事眼底病及青光眼方面的研究

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Cancers (Basel), 2021, 13(22): 5862., articleTitle=Tylectomy safety in salvage of eyes with retinoblastoma, refAbstract=null)], funds=null, companyList=[AuthorCompany(id=1199335054175469675, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, xref=1, ext=[AuthorCompanyExt(id=1199335054188052588, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054175469675, language=EN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=1Department of Ophthalmology, School of Clinical Medicine, School of Public Health, Weifang Medical University, Weifang, Shandong 261053, China), AuthorCompanyExt(id=1199335054192246893, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054175469675, language=CN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=1潍坊医学院临床医学院眼科学教研室，山东潍坊 261053)]), AuthorCompany(id=1199335054288715893, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, xref=2, ext=[AuthorCompanyExt(id=1199335054292910198, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054288715893, language=EN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=2Senior Department of Ophthalmology, the Third Medical Center of PLA General Hospital, Beijing 100039, China), AuthorCompanyExt(id=1199335054301298807, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054288715893, language=CN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=2解放军总医院第三医学中心眼科医学部，北京 100039)]), AuthorCompany(id=1199335054372601982, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, xref=3, ext=[AuthorCompanyExt(id=1199335054380990591, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054372601982, language=EN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=3Department of Health Statistics, School of Public Health, Weifang Medical University, Weifang, Shandong 261053, China), AuthorCompanyExt(id=1199335054385184896, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, companyId=1199335054372601982, language=CN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=3潍坊医学院公共卫生学院卫生统计学教研室，山东潍坊 261053)])], figs=[ArticleFig(id=1199335058202001703, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=EN, label=Fig.1, caption=Comparison of baseline data of retinoblastoma patients before and after weighting by the propensity score IPTW method, figureFileSmall=hw/2Fki9Mpucd9ceXMioTA==, figureFileBig=n7BELv2nhQand6hkx3oJFA==, tableContent=null), ArticleFig(id=1199335058290082096, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=CN, label=图1, caption=倾向性评分IPTW加权前后视网膜母细胞瘤患者基线资料对比

IPTW. 逆概率加权法

, figureFileSmall=hw/2Fki9Mpucd9ceXMioTA==, figureFileBig=n7BELv2nhQand6hkx3oJFA==, tableContent=null), ArticleFig(id=1199335058558517564, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=EN, label=Fig.2, caption=Kaplan-Meier survival curves of retinoblastoma patients in three groups before and after weighting by the propensity score IPTW method, figureFileSmall=yX6pAbVfJTqWglHcg+W79g==, figureFileBig=sB2eOa8yczlTs+lXeCK8wA==, tableContent=null), ArticleFig(id=1199335058688540998, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=CN, label=图2, caption=倾向性评分IPTW加权前(A)、后(B)3组视网膜母细胞瘤患者Kaplan-Meier生存曲线

IPTW. 逆概率加权法

, figureFileSmall=yX6pAbVfJTqWglHcg+W79g==, figureFileBig=sB2eOa8yczlTs+lXeCK8wA==, tableContent=null), ArticleFig(id=1199335058877284686, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=EN, label=Tab.1, caption=

Comparison of baseline data of retinoblastoma patients with three treatment modalities [n(%)]

, figureFileSmall=null, figureFileBig=null, tableContent=
变量手术组(n=426)化疗组(n=200)手术联合化疗组(n=562)P
IPTW加权前IPTW加权后
年龄<0.0010.837
<1岁129(30.3)128(64.0)289(51.4)
≥1岁297(69.7)72(36.0)273(48.6)
性别0.1110.732
女203(47.7)109(54.5)282(50.2)
男223(52.3)91(45.5)280(49.8)
种族0.2680.768
黑种人65 (15.3)21 (10.5)82(14.6)
白种人309(72.5)154(77.0)402(71.5)
其他或未知52(12.2)25(12.5)78(13.9)
多灶性0.1630.297
1个病灶419(98.4)195(97.5)545(97.0)
≥2个病灶7(1.6)5(2.5)17(3.0)
是否为首要恶性肿瘤0.1980.091
否3(0.7)01(0.2)
是423(99.3)200(100.0)561(99.8)
眼别<0.0010.890
左眼208(48.8)41(20.5)140(24.9)
右眼188(44.1)62(31.0)145(25.8)
双眼30(7.1)97(48.5)277(49.3)
), ArticleFig(id=1199335059007308118, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=CN, label=表1, caption=

3种治疗方式的视网膜母细胞瘤患者基线资料比较 [例(%)]

, figureFileSmall=null, figureFileBig=null, tableContent=
变量手术组(n=426)化疗组(n=200)手术联合化疗组(n=562)P
IPTW加权前IPTW加权后
年龄<0.0010.837
<1岁129(30.3)128(64.0)289(51.4)
≥1岁297(69.7)72(36.0)273(48.6)
性别0.1110.732
女203(47.7)109(54.5)282(50.2)
男223(52.3)91(45.5)280(49.8)
种族0.2680.768
黑种人65 (15.3)21 (10.5)82(14.6)
白种人309(72.5)154(77.0)402(71.5)
其他或未知52(12.2)25(12.5)78(13.9)
多灶性0.1630.297
1个病灶419(98.4)195(97.5)545(97.0)
≥2个病灶7(1.6)5(2.5)17(3.0)
是否为首要恶性肿瘤0.1980.091
否3(0.7)01(0.2)
是423(99.3)200(100.0)561(99.8)
眼别<0.0010.890
左眼208(48.8)41(20.5)140(24.9)
右眼188(44.1)62(31.0)145(25.8)
双眼30(7.1)97(48.5)277(49.3)
), ArticleFig(id=1199335059170885977, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=EN, label=Tab.2, caption=

Multifactorial Cox regression analysis of retinoblastoma patients outcomes before and after propensity score weighting for the three treatment modalities (n=1188)

, figureFileSmall=null, figureFileBig=null, tableContent=
纳入变量IPTW加权前IPTW加权后
HR(95%CI)PHR(95%CI)P
年龄
<1岁参照参照
≥1岁0.967(0.853~1.096)0.5971.061(0.911~1.236)0.448
性别
女参照参照
男0.855(0.760~0.962)0.0090.828(0.714~0.960)0.012
种族
黑种人参照参照
白种人1.058 (0.890~1.258)0.5251.079(0.868~1.341)0.495
其他或未知1.101(0.878~1.382)0.4041.196(0.907~1.577)0.205
多灶性
1个病灶参照参照
≥2个病灶0.464(0.282~0.762)0.0020.399(0.268~0.594)<0.001
是否为首要恶性肿瘤
否参照参照
是0.425(0.141~1.280)0.1280.370(0.201~0.682)0.001
眼别
左眼参照参照
右眼0.926(0.802~1.070)0.2980.931(0.781~1.111)0.431
双眼0.875(0.743~1.030)0.1090.915(0.748~1.119)0.387
治疗方式
手术参照参照
化疗1.375(1.138~1.662)<0.0011.367(1.100~1.700)0.005
手术联合化疗1.232(1.067~1.424)0.0051.132(0.963~1.330)0.133
), ArticleFig(id=1199335060328513888, tenantId=1146029695717560320, journalId=1189873630562394117, articleId=1199335052925567024, language=CN, label=表2, caption=

倾向性评分IPTW加权前后视网膜母细胞瘤3种治疗方式疗效的多因素Cox回归分析结果(n=1188)

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纳入变量IPTW加权前IPTW加权后
HR(95%CI)PHR(95%CI)P
年龄
<1岁参照参照
≥1岁0.967(0.853~1.096)0.5971.061(0.911~1.236)0.448
性别
女参照参照
男0.855(0.760~0.962)0.0090.828(0.714~0.960)0.012
种族
黑种人参照参照
白种人1.058 (0.890~1.258)0.5251.079(0.868~1.341)0.495
其他或未知1.101(0.878~1.382)0.4041.196(0.907~1.577)0.205
多灶性
1个病灶参照参照
≥2个病灶0.464(0.282~0.762)0.0020.399(0.268~0.594)<0.001
是否为首要恶性肿瘤
否参照参照
是0.425(0.141~1.280)0.1280.370(0.201~0.682)0.001
眼别
左眼参照参照
右眼0.926(0.802~1.070)0.2980.931(0.781~1.111)0.431
双眼0.875(0.743~1.030)0.1090.915(0.748~1.119)0.387
治疗方式
手术参照参照
化疗1.375(1.138~1.662)<0.0011.367(1.100~1.700)0.005
手术联合化疗1.232(1.067~1.424)0.0051.132(0.963~1.330)0.133
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基于倾向性评分逆概率加权法的视网膜母细胞瘤疗效评价及其预后影响因素分析
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石丽娟 1, 2 , 李丽 1, 2 , 石福艳 3 , 周希彬 2 , 吴志鸿 2, *
解放军医学杂志 | 临床研究 2024,49(3): 302-307
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解放军医学杂志 | 临床研究 2024, 49(3): 302-307
基于倾向性评分逆概率加权法的视网膜母细胞瘤疗效评价及其预后影响因素分析
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石丽娟1, 2, 李丽1, 2, 石福艳3, 周希彬2, 吴志鸿2, *
作者信息
  • 1潍坊医学院临床医学院眼科学教研室，山东潍坊 261053
  • 2解放军总医院第三医学中心眼科医学部，北京 100039
  • 3潍坊医学院公共卫生学院卫生统计学教研室，山东潍坊 261053

    • 石丽娟，硕士研究生，主要从事眼底病及青光眼方面的研究

通讯作者:

吴志鸿，E-mail：
Efficacy evaluation and prognostic factors analysis of retinoblastoma based on propensity score inverse probability weighting method
Li-Juan Shi1, 2, Li Li1, 2, Fu-Yan Shi3, Xi-Bin Zhou2, Zhi-Hong Wu2, *
Affiliations
  • 1Department of Ophthalmology, School of Clinical Medicine, School of Public Health, Weifang Medical University, Weifang, Shandong 261053, China
  • 2Senior Department of Ophthalmology, the Third Medical Center of PLA General Hospital, Beijing 100039, China
  • 3Department of Health Statistics, School of Public Health, Weifang Medical University, Weifang, Shandong 261053, China
出版时间: 2024-03-28 doi: 10.11855/j.issn.0577-7402.2030.2023.0630
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摘要
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目的 评估手术、化疗及手术联合化疗3种治疗方式对视网膜母细胞瘤(RB)的疗效，分析RB患者的预后影响因素。方法 选取2000年1月1日-2019年12月31日美国监测、流行病学和最终结果(SEER)数据库登记的RB患者1188例临床资料进行回顾性分析。采用倾向性评分逆概率加权法(IPTW)对接受手术、化疗或手术联合化疗治疗的3组患者基线特征进行平衡。采用Log-rank检验比较3组患者的生存概率，采用Cox回归模型分析RB患者预后影响因素。结果 共纳入RB 1188例，其中手术组426例，化疗组200例，手术联合化疗组562例。经IPTW加权后，患者的年龄、性别、种族等基线资料均达到平衡(P>0.05)。Log-rank检验结果显示，加权前后3组生存曲线比较差异均有统计学意义(P<0.05)；加权后，手术组患者生存情况明显优于化疗组和手术联合化疗组(P<0.05)，化疗组与手术联合化疗组比较差异无统计学意义(P>0.05)。加权后患者1年、3年和5年的生存概率为：手术组99.7%、98.9%和98.6%；化疗组97.4%、95.8%和95.8%；手术联合化疗组97.9%、95.8%和95.0%。Cox回归分析结果显示，与手术组比较，化疗组患者特异性死亡风险比为1.367(95%CI 1.100~1.700)，手术联合化疗组特异性死亡风险比为1.132(95%CI 0.963~1.330)；与RB病灶为1个的患者比较，RB病灶数≥2个的患者特异性死亡风险比为0.399(95%CI 0.268~0.594)。结论 RB患者接受治疗后生存概率较高；在控制年龄、性别等因素的影响后，3种治疗方式中手术的疗效更好；多灶性可能是RB患者预后的独立影响因素。

视网膜母细胞瘤  /  逆概率加权  /  疗效  /  生存分析

Objective To evaluate the efficacy of surgery, chemotherapy and surgery combined chemotherapy for retinoblastoma (RB), and analyze the prognostic factors of RB patients. Methods Clinical data of 1188 RB patients registered in the Surveillance, Epidemiology and End Results (SEER) database from January 2000 to December 2019 were retrospectively analyzed. The baseline characteristics of patients treated with surgery, chemotherapy or surgery combined with chemotherapy were balanced by inverse probability of treatment weighting (IPTW). Log-rank test analysis was used to compare the survival probability of patients in the 3 groups, and Cox regression models were used to analyse the factors influencing the prognosis of RB patients. Results A total of 1188 RB cases were included in this study, including 426 cases in surgery group, 200 cases in chemotherapy group and 562 cases in surgery combined with chemotherapy group. After IPTW weighting, baseline data such as age, sex and race were balanced (P>0.05). Log-rank test results showed that the survival curves of the three groups were significantly different before and after weighting (P<0.05). After weighted, the survival of patients in surgery group was significantly better than that in chemotherapy group and surgery combined chemotherapy group (P<0.05), and there was no statistical significance between chemotherapy group and surgery combined chemotherapy group (P>0.05).The weighted patient survival probability at 1st, 3rd and 5th years were 99.7%, 98.9% and 98.6% in surgery group; 97.4%, 95.8% and 95.8% in chemotherapy group; and 97.9%, 95.8% and 95.0% in surgery combined chemotherapy group. Cox regression analysis showed that compared with surgery group, the specific risk ratio of death was 1.367 (95%CI 1.100-1.700) in chemotherapy group and 1.132 (95%CI 0.963-1.330) in combined chemotherapy group. Compared with patients with 1 RB lesion, the patient-specific mortality risk ratio for patients with 2 or more RB lesions was 0.399 (95%CI 0.268-0.594). Conclusions Patients with RB have higher survival rates probability after treatment. After controlling the influence of age, sex and other factors, the effect of surgery was better among the three treatment methods. Multifocality may be an independent prognostic factor in RB patients.

retinoblastoma  /  inverse probability of treatment weighting  /  efficacy  /  survival analysis
石丽娟, 李丽, 石福艳, 周希彬, 吴志鸿. 基于倾向性评分逆概率加权法的视网膜母细胞瘤疗效评价及其预后影响因素分析. 解放军医学杂志, 2024 , 49 (3) : 302 -307 . DOI: 10.11855/j.issn.0577-7402.2030.2023.0630
Li-Juan Shi, Li Li, Fu-Yan Shi, Xi-Bin Zhou, Zhi-Hong Wu. Efficacy evaluation and prognostic factors analysis of retinoblastoma based on propensity score inverse probability weighting method[J]. Medical Journal of Chinese People’s Liberation Army, 2024 , 49 (3) : 302 -307 . DOI: 10.11855/j.issn.0577-7402.2030.2023.0630
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视网膜母细胞瘤(retinoblastoma，RB)是婴儿期和儿童常见的眼部恶性肿瘤[1]。全球每年约有8000例儿童被确诊为RB，中国每年约有1100例新发RB患者[2-3]。斜视和“白眼”是RB的两个主要表现。RB如果未在早期阶段得到治疗，往往造成严重后果，如视力丧失，甚至死亡[4]。传统的RB治疗方法包括手术(眼球摘除术)、化学药物治疗(化疗)、放射治疗、局部治疗等[5]。近年来，基因治疗、玻璃体手术治疗也开始应用[6]。随着医疗技术的进步，RB的治疗原则变更为在确保患儿生命及保留眼球的基础上，尽可能保留一部分视功能，以提高其生存质量[3]。目前由于病例临床信息采集困难、缺乏足够的病例等原因，RB治疗方式与预后的评价研究相对较少。本研究基于美国监测、流行病学和最终结果(Surveillance, Epidemiology, and End Results，SEER)数据库中RB患者临床数据的回顾性分析，评估RB经手术、化疗和手术联合化疗治疗后的生存情况，并分析与该病预后相关的临床特征。相关研究显示，RB患者的分期、多灶性及相关临床特征会影响疗效和预后，若不校正各变量之间的混杂因素影响，则可能与临床实际结果冲突[3,7]。本研究采用倾向性评分逆概率加权法(inverse probability of treatment weighting，IPTW)对RB患者基线资料进行平衡校正，以提供各变量间或各组间更公平的比较，以期得到与临床更为贴切的结果[8-10]，为临床评估RB患者病情和制定治疗方案提供参考。
1 资料与方法
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1.1 研究对象
基于美国SEER的肿瘤患者数据库(http://seer.cancer.gov/)，对该数据库中2000年1月1日-2019年12月31日登记的1188例RB患者的数据进行回顾性分析。纳入标准：(1)诊断时间为2000-2019年；(2)依据国际疾病分类(International Classification of Diseases，ICD)第10版(恶性肿瘤分类标准)查询视网膜(C692)和国际疾病肿瘤学分类(International Classification of Diseases for Oncology，ICD-O)第3版形态学代码查询母细胞瘤(9510-3)；(3)接受手术治疗、化疗或手术联合化疗。排除标准：临床信息不完整或不明确；患者生存状况不明或死于其他原因。
1.2 方法
1.2.1 资料收集
提取符合标准的1188例RB患儿的所有基线资料，包括种族、性别、诊断年龄、眼别、是否为首要恶性肿瘤、多灶性、治疗方式、生存时间、生存状态等；将所有患者按照治疗方式分为3组：手术组(426例)、化疗组(200例)和手术联合化疗组(562例)。
1.2.2 临床指标分析
(1)本研究的起始日期为2000年1月1日，截止日期为2019年12月31日；设定终点事件为RB患者死亡。(2)对比分析3组患者的一般基线资料，包括年龄、性别、种族、是否为首要恶性肿瘤和眼别。基线资料的差异性采用IPTW法校正，该方法参考使用了Ridgeway等[9]研发的R软件中的Twang包及其相关研究[11-12]。(3)3种治疗方式的生存疗效采用Kaplan-Meier生存曲线描述，并采用Log-rank检验比较各组生存率，通过计算各组RB患者的生存率评估各治疗方式的疗效。(4)根据双稳健模型分析理念，IPTW法虽可纠正绝大多数的不平衡性，但有时会出现不完全纠正的情况，故RB患者预后影响因素分析进一步采用Cox回归模型分析，以实现双稳健多因素分析[13]。
1.3 统计学处理
采用R4.0.3软件和IBM SPSS Statistics 26软件进行统计分析。计量资料以$\bar{x}±s$表示，计数资料以率(%)表示；基线资料的差异性采用IPTW法校正；生存资料采用Kaplan-Meier生存曲线描述，并采用Log-rank检验比较各组生存率；采用Cox回归模型分析患者预后影响因素。P<0.05为差异有统计学意义。
2 结果
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2.1 3组RB患者基线资料比较
1188例RB患者中，手术426例，化疗200例，手术与化疗联合治疗562例。在IPTW加权前，3组患者除年龄、眼别分布差异有统计学意义(P<0.05)外，其他变量比较差异均无统计学意义(P>0.05)；经IPTW加权后，3组患者各基线资料比较差异均无统计学意义(P>0.05)，所有基线资料均达到平衡(表1，图1)。
2.2 3组RB患者加权前后生存情况比较
Log-rank检验结果显示，加权前后3组RB患者的生存曲线比较差异均有统计学意义(P<0.05)。加权前，手术组与化疗组比较差异无统计学意义(P>0.05)，手术组生存情况明显优于手术联合化疗组(P<0.05)，化疗组与手术联合化疗组比较差异无统计学意义(P>0.05)；加权后，手术组生存情况明显优于化疗组和手术联合化疗组(P<0.05)，化疗组与手术联合化疗组比较差异无统计学意义(P>0.05，图2)。对3组RB患者加权前后的生存概率进行分析，结果显示，倾向性评分IPTW法加权前，手术组患者1年、3年与5年的生存概率分别为99.5%、98.7%和98.0%；化疗组患者1年、3年与5年的生存概率分别为97.4%、96.0%和94.9%；手术联合化疗组患者1年、3年、5年的生存概率分别为98.3%、95.9%和95.1%。IPTW法加权后，手术组患者1年、3年与5年的生存概率分别为99.7%、98.9%和98.6%；化疗组患者1年、3年与5年的生存概率分别为97.4%、95.8%和95.8%；手术联合化疗组患者1年、3年、5年的生存概率分别为97.9%、95.8%和95.0%。
2.3 IPTW加权后RB患者预后的多因素分析
在校正种族、性别、诊断年龄、眼别、是否为首要恶性肿瘤、多灶性、治疗方式等变量后，对3组RB患者死亡风险对比分析结果显示，与RB病灶数为1个的患者比较，RB病灶数≥2个的患者特异性死亡风险比为0.399(95%CI 0.268~0.594)。与手术组患者比较，化疗组患者特异性死亡风险比为1.367(95%CI 1.100~1.700)，手术联合化疗组特异性死亡风险比为1.132(95%CI 0.963~1.330，表2)。
3 讨论
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SEER数据库是一个以区域人群为基础的癌症报告系统，包括美国特定地理区域内的所有癌症病例，涵盖了28%的美国人口，可用于1974年至今的癌症诊断、治疗和生存的纵向趋势分析，特别适用于对特定亚群和罕见或惰性癌症类型的纵向研究[14]。我国的RB病例数居全球第一，约占全球总数的1/6。RB恶性程度增高，占儿童致盲原因的5%，如得不到治疗则病死率较高，但若早期接受系统治疗，其生存率可达95%。RB患者常见死亡原因为延误治疗时机导致眼外扩散或继发肿瘤[15]。近年来RB的诊疗取得明显进展，但仍具有一定的挑战性，因此，研究RB的治疗策略具有临床实际意义[16]。本研究基于SEER数据库，比较RB手术、化疗和手术联合化疗3种治疗方式的疗效，以期为RB治疗相关的临床决策提供参考。由于SEER数据库整体数据较为丰富，数据跨度时间较长，且部分数据缺失，因此，本研究选择种族、性别、诊断年龄、眼别、是否为首要恶性肿瘤、多灶性、治疗方式、生存时间、生存状态及随访信息完整的RB患者，以提高研究结论的准确性。对RB患者的临床数据，采用IPTW计算倾向性评分，使变量达到事后随机化。
本研究结果显示，RB患者接受治疗后的生存率较高，与Baker等[17]和Temming等[18]的研究结果一致。
本研究多因素Cox回归分析结果显示，在校正了诊断年龄、性别等因素后，RB手术治疗的疗效优于化疗和手术联合化疗；多灶性为RB患者预后的独立影响因素。其中，化疗组患者的特异性死亡风险是手术组的1.367倍，手术联合化疗组患者的特异性死亡风险是手术组的1.132倍，提示手术治疗的疗效优于化疗和手术联合化疗。该结果与Zhao等[19]的结果一致。本研究结果显示，与RB病灶为1个患者的比较，RB病灶数≥2个的患者特异性死亡风险比为0.399(95%CI 0.268~0.594)。该结果提示多灶性为RB患者预后的保护性因素，与Kaliki等[7]研究结果不一致。究其原因，可能与本研究纳入的多灶性RB患者数量较少有关，故有关多灶性与RB预后间的关系尚待进一步研究。
综上所述，本研究结果显示：(1)RB患者接受治疗后生存率较高；(2)在校正年龄、性别等因素的影响后，3种治疗方式中手术治疗的疗效更好；(3)多灶性可能是RB患者预后的独立影响因素。
本研究存在一定的局限性：(1)基于SEER公共数据库进行数据分析，部分重要的影响因素在SEER数据库中并无记录，如RB分期。有研究显示，对国际分期A期、B期、C期的早期RB患儿治疗效果较好，而国际分期D期、E期的晚期患儿保眼率较低[3]。因此，对于分期对RB患者预后的影响，后期仍需病例资料更为齐全的数据对研究进行优化。(2)由于SEER数据库中对化疗方案和手术方式未做详细说明，因此无法对此内容做进一步比较。后期有条件的情况下，将对RB的治疗方式与预后之间的关系做进一步探讨。(3)本研究未进行外部验证，后续需要大样本队列数据进行验证。
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doi: 10.11855/j.issn.0577-7402.2030.2023.0630
  • 接收时间：2022-09-28
  • 首发时间：2025-11-23
  • 出版时间：2024-03-28
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  • 收稿日期：2022-09-28
  • 录用日期：2022-11-27
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    1潍坊医学院临床医学院眼科学教研室，山东潍坊 261053
    2解放军总医院第三医学中心眼科医学部，北京 100039
    3潍坊医学院公共卫生学院卫生统计学教研室，山东潍坊 261053

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鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
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