中国循证儿科杂志 ›› 2021, Vol. 16 ›› Issue (2): 104-108.DOI: 10.3969/j.issn.1673-5501.2021.02.005

• 论著 • 上一篇    下一篇

单眼眼内期视网膜母细胞瘤168例疗效及生存质量分析

周宇晨, 赵军阳, 张成玥, 洪慧敏, 赵文, 于彤, 金眉, 马晓莉   

  1. 首都医科大学附属北京儿童医院 北京,100045
  • 收稿日期:2020-05-18 修回日期:2021-04-02 出版日期:2021-04-25 发布日期:2021-06-04
  • 通讯作者: 马晓莉,email:mxl1123@vip.sina.com;金眉,email:meijin1975@sina.com

Analysis of the curative effect and quality of life of 168 cases of unilateral intraocular retinoblastoma

ZHOU Yuchen, ZHAO Junyang, ZHANG Chengyue, HONG Huimin, ZHAO Wen, YU Tong, JIN Mei, MA Xiaoli   

  1. Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
  • Received:2020-05-18 Revised:2021-04-02 Online:2021-04-25 Published:2021-06-04
  • Contact: MA Xiaoli, email: mxl1123@vip.sina.com; JIN Mei, email: meijin1975@sina.com

摘要: 背景 视网膜母细胞瘤(Rb)是儿童期最常见的恶性肿瘤,在90% Rb患儿所在的发展中国家,减少死亡仍是一个挑战,且患儿的生存质量较少受到关注。目的 总结单眼眼内期Rb患儿疗效、安全性及生存质量,为优化治疗方案和提高治疗安全性及有效性提供证据。设计 回顾性非随机对照研究。方法 回顾性分析2009年11月6日至2019年9月5日首都医科大学附属北京儿童医院收治的单眼眼内期Rb患儿的临床资料,末次随访日期为2020年4月20日。化疗方案为根据加拿大多中心RB 2003方案改良的北京儿童医院RB-2009方案,并应用810 nm及532 nm激光、冷凝器等技术进行局部治疗。分析保眼率、生存率、死亡原因、不良反应等,并对随访时≥5岁儿童采用儿童生存质量普适性核心量表和儿童生存质量癌症模块量表进行生存质量调查。主要结局指标 5年总生存率(OS)和生存质量。结果 符合纳入标准的6 262例Rb,完成随访168例,中位随访时间51(8~125)月,平均化疗疗程4.19±2.16(1~12)疗程。B期1例,C期3例,D期95例,E期69例。共眼球摘除89只(53.0%),其中D期42例,E期47例;直接眼球摘除30例(33.7%),化疗后眼球摘除59例(66.3%)。成功保眼79例(47.0%),其中D期53例(55.79%),E期22例(31.9%)。预计全部患儿5年总生存率(OS)为95.2%。眼球摘除和保眼治疗患儿5年OS分别为95.5%和94.9%,差异无统计学意义(P=0.78)。5例(3.0%)发生可逆性Ⅰ级听力损害,无Ⅱ~Ⅳ级听力损害发生;所有患儿均发生骨髓抑制,6例次(3.6%)输注PLT,无感染相关死亡发生。106例≥5岁患儿完成生存质量调查,眼球摘除患儿对外貌的自我感觉维度、社会维度和角色维度平均得分均低于保眼患儿,差异均有统计学意义(P<0.05)。死亡8例,D期5例,E期3例,2例因经济原因放弃治疗后死亡,6例复发后颅内转移死亡。结论 VEC(长春新碱、依托泊苷和卡铂)方案全身化疗结合局部治疗对单眼眼内期Rb患儿是安全、有效的。颅内转移仍然是Rb相关死亡最常见的原因。≥5岁眼球摘除患儿在外貌自我感觉、社会、角色维度生存质量下降。

关键词: 视网膜母细胞瘤, 生存率, 生存质量

Abstract: Background Retinoblastoma(Rb) is a common malignant tumor in childhood. In developing countries where 90% of children with Rb are located, reducing deaths is still a challenge, and the quality of life of children has received little attention.Objective To summarize the efficacy, safety and quality of life of children with unilateral intraocular retinoblastoma, and provide evidence for optimizing the treatment plan and improving the safety and effectiveness of treatment.DesignRetrospective non-randomized controlled study.Methods To retrospectively analyze the clinical data of children with unilateral intraocular retinoblastoma admitted to Beijing Children's Hospital, Capital Medical University from November 6, 2009 to September 5, 2019. The last follow-up date was April 20, 2020. The chemotherapy program was the RB-2009 program of Beijing Children's Hospital, which was improved according to the Canadian multi-center RB 2003 program, and 810 nm and 532 nm laser, condenser and other technologies were used for local treatment. The eye-preservation rate, survival rate, cause of death, toxic and side effects, etc. were analyzed, and the Child Quality of Life Universal Scale and the Child Quality of Life Cancer Module Scale were used to conduct quality of life surveys for children older than 5 years of age.Main outcome measures Five year overall survival(OS) rate and life quality.Results A total of 6,262 cases of Rb were included and 168 patients were followed up for 8 to 125 months (median time 51 months). There were 1 case in stage B, 3 cases in stage C, 95 cases in stage D, and 69 cases in stage E and the total course of chemotherapy was 1 to 12 cycles(mean 4.2± 2.2 courses).Enucleation was performed in 89 (53.0%) with 42 cases in stage D and 47 in stage E. Among them, 30 cases (33.7%) were directly enucleated, and 59 cases (66.3%) were enucleated after chemotherapy. Seventy-nine cases (47.0%) were preserved with 53(55.79%) in stage D, and 22 (31.9%) in stage E. The 5-year expected OS of all children was 95.2%. The expected 5-year OS was 95.5% in children with eye enucleation and 94.9% in children with preservation treatment. The differences were not statistically significant(P=0.78). Five patients (3.0%) suffered from reversible grade Ⅰ hearing impairment, and no grade Ⅱ-Ⅳ hearing impairment occurred. All children developed bone marrow suppression, and 6 patients received platelet transfusion (3.6%), and no infection-related death occurred. Among 106 patients completing the quality of life survey, the score of children with enucleation was lower than that of children with eye preservation in questions about self-perception, society and role. The differences were statistically significant(P<0.05).There were 8 deaths with 5 cases in stage D and 4 cases in stage E, 2 of which died after giving up treatment due to economic reasons, and 6 of which died of intracranial metastasis after relapse.Conclusion VEC (Vincristine, Etoposide and Carboplatin) regimen combined with systemic chemotherapy and local treatment is safe and effective for children with unilateral intraocular retinoblastoma. Intracranial metastasis remains the most common cause of Rb-related death. The results of a survey on the quality of life of children over 5 years of age show that the eye enucleation children have a reduced quality of life in terms of appearance, self-feeling, society and role.

Key words: Retinoblastoma, Survival rate, Quality of life