Objective: To summarize the clinical characteristics, treatment response and prognostic factors of rhabdomyosarcoma (RMS) in children. Methods: The clinical characteristics such as age at diagnosis, primary tumor site, tumor size, pathological type, clinical stage, and risk grouping of 213 RMS patients (140 males and 73 females) treated in Hematology Oncology Center of Beijing Children's Hospital, Capital Medical University, from May 2006 to June 2018 were analyzed retrospectively. The clinical characteristics, overall survival (OS), event free survival (EFS) and prognostic factors of children treated with the Beijing Children's Hospital-Rhabdomyosarcoma (BCH-RMS) regimen were analyzed. Survival data were analyzed by Kaplan-Meier survival analysis, and single factor analysis was performed by Log-Rank test. Results: The diagnostic age of 213 cases was 48.0 months (ranged 3.0-187.5 months), of which 136 cases (63.8%) were younger than 10 years old. The head and neck region was the most common primary site of tumor (30%, 64 cases), followed by the genitourinary tract (26.8%, 57 cases). Among pathological subtypes, embryonal RMS accounted for 71.4% (152 cases), while alveolar RMS and anaplastic RMS accounted for only 26.8% (57 cases) and 1.9% (4 cases), respectively. According to the Intergroup Rhabdomyosarcoma Study Group (IRS), IRS-Ⅲ and Ⅳ accounted for 85.0% (181 cases) of all RMS patients. In all patients, 9.4% (20 cases) patients were divided in to low-risk group, 52.1% (111 cases) patients in to intermediate -risk group, 25.8% (55 cases) patients in to high-risk group, and 12.7% (27 cases) patients in to the central nervous system invasion group, respectively. All patients with RMS received chemotherapy. The cycles of chemotherapy were 13.5 (ranged 5.0-18.0) for patients without event occurrence, while 14.2 (ranged 3.0-30.0) for patients with event occurrence. Among the 213 patients, 200 patients had surgical operation, of whom 103 patients underwent surgery before chemotherapy and 97 patients at the end of chemotherapy, 21 patients had secondary surgical resection. Radiotherapy was performed in 114 patients. The follow-up time was 23.0 months (ranged 0.5-151.0 months) . There were 98 patients with relapsed or progressed disease and 67 patients with death. The median time to progression was 10 months, of which 67 (68.4%) relapse occurred within 1 year and no recurrence occurred after follow-up for more than 5 years. The 3-year EFS and 5-year EFS were (52±4) % and (48±4) %, while the 3-year OS and 5-year OS were (65±4) % and (64±4) % by survival analysis. The 5-year OS of the low-risk, intermediate-risk, the high-risk were 100%, (74±5) %, (48±8) %, and the 2-year OS of the central nervous system invasion group was (36±11) % (χ(2)=33.52, P<0.01). The 5-year EFS of the low-risk, intermediate-risk, the high-risk were (93±6) %, (51±5) %, (36±7) % and the 2-year EFS of the central nervous system invasion group was (31±10) % (χ(2)=24.73, P<0.01) . Survival factor analysis suggested that the OS of children was correlated with age(χ(2)=4.16, P=0.038), tumor TNM stage (χ(2)=22.02, P=0.001), IRS group (χ(2)=4.49, P<0.01) and the risk group (χ(2)=33.52, P<0.01). Conclusions: This study showed that the median age of newly diagnosed RMS patients was 4 years. The head and neck and the genitourinary tract were the most common primary origin of RMS. The OS was low in single-center RMS children. The median time to recurrence was 10 months, and recurrence was rare 3 years later.
目的： 总结横纹肌肉瘤（RMS）患儿的临床特征、治疗效果，分析影响预后的相关因素。 方法： 回顾性分析2006年5月至2018年6月在首都医科大学附属北京儿童医院诊治的RMS患儿213例（男140例、女73例）的初诊时年龄、原发瘤灶部位、瘤灶大小、病理分型、临床分期、危险度分组等临床特征。并对规律治疗随访的患儿进行总生存率（OS）、无事件生存率（EFS）及预后影响因素的分析。生存资料采用Kaplan-Meier生存分析法，单因素分析采用Log-Rank检验。 结果： 213例患儿诊断年龄48.0（3.0~187.5）月龄，其中136例患儿<10岁（63.8%）。原发瘤灶以头颈部最常见（64例，30.0%），其次为泌尿生殖系统（57例，26.8%）。病理亚型中胚胎型152例（71.4%），腺泡型57例（26.8%），多形型或间变型4例（1.9%）。IRS分期Ⅲ和Ⅳ期患儿181例（85.0%）。低危组20例（9.4%），中危组111例（52.1%），高危组55例（25.8%），中枢侵犯组27例（12.7%）。所有患儿均进行了化疗，其中无事件患儿化疗13.5（5.0~18.0）个疗程，复发进展患儿化疗14.2（3.0~30.0）个疗程。200例患儿进行手术治疗，其中化疗前手术103例，化疗后手术97例，二次手术者21例。114例患儿进行了放疗。213例患儿随访时间为23.0（0.5~151.0）个月，其中98例复发或进展，中位进展时间10个月，67例死亡。67例（68.4%）复发进展时间在1年内，随访大于5年患儿均无复发。生存分析显示RMS患儿3年和5年EFS分别为（52±4）%、（48%±4）%，3年和5年OS分别为（65±4）%和（64±4）%。低危组、中危组和高危组5年OS分别为100%、（74±5）%、（48±8）%，中枢侵犯组2年OS为（36±11）%（χ(2)=33.52，P<0.01）；低危组、中危组和高危组5年EFS分别为（93±6）%、（51±5）%、（36±7）%，中枢侵犯组2年EFS为（31±10）%（χ(2)=24.73，P<0.01）。生存因素分析提示患儿的OS与年龄（χ(2)=4.16，P=0.038）、肿瘤TNM分期（χ(2)=22.02，P=0.001）、IRS术后分期（χ(2)=4.49，P<0.01）以及危险度分组（χ(2)=33.52，P<0.01）有相关性。 结论： 单中心较大样本RMS中位年龄4岁；原发瘤灶以头颈部和泌尿生殖系统为多见，患儿OS偏低。复发进展中位时间10个月，3年后复发少见。.
Keywords: Child; Follow-up studies; Prognosis; Rhabdomyosarcoma.