*Department of Pediatric Surgery and Pediatric Urology, University Children's Hospital, Tuebingen, Germany †Department of Pediatric Hematology, Oncology and Immunology, Olgahospital, Stuttgart, Germany ‡Department of Clinical Epidemiology and Applied Biometry, University of Tuebingen, Tuebingen, Germany §Department for Pediatric Hematology and Oncology, University Hospital, Frankfurt/Main, Germany ¶Department of Paidopathology, University Hospital, Kiel, Germany ‖Department of Radiotherapy, University Hospital, Muenster, Germany **Department of Oncology, University Children's Hospital, Zurich, Switzerland.
Ann Surg. 2014 Jun;259(6):1166-72. doi: 10.1097/SLA.0b013e3182a6f320.
To analyze the clinical course, treatment, complications, outcome, and quality of life (QOL) in patients with perineal/perianal rhabdomyosarcoma (PRMS) treated within the CWS-86, -91, -96, and -2002P trials.
Although multiple international study trials exist for the treatment of rhabdomyosarcoma, only very limited information is given on treatment, outcome, and QOL in PRMS.
A total of 35 patients suffering from PRMS were treated with neoadjuvant chemotherapy. Local therapy with radiation and/or surgery was performed, followed by adjuvant chemotherapy. Functional long-term follow-up was evaluated by a gastrointestinal/QOL survey.
Thirty-two patients were evaluated (exclusion n = 3). Eight patients had embryonal histology, and 24 patients had alveolar histology. The median age was 108 months (median follow-up: 5.8 years). The 5-year overall survival was 47% (95% confidence interval: 29-64). Sixteen IRS (Intergroup Rhabdomyosarcoma Study) III and IV patients had locoregional lymph node involvement at diagnosis. Seven patients were treated with chemotherapy/surgery alone [5-year event-free survival (EFS): 85.7%]. Eleven patients received only radiochemotherapy (5-year EFS: 27.3%). Combined radiochemotherapy/surgery was used in 12 patients (5-year EFS: 63.6%). Two patients were treated only with chemotherapy and they died. Patients with embryonal histology had a significantly better 5-year EFS (87.5%) than patients with alveolar histology (39.1%; P = 0.013). Some patients reported symptoms of fecal incontinence. The median Wexner fecal incontinence score was 9 (possible range: 0-20), and the median QOL score was 90.5 (applicable range: 0-144).
The outcome of these patients remains unsatisfactory. Prognostic factors for a favorable outcome are tumor size of smaller than 5 cm, negative locoregional lymph nodes, age less than 10 years, low IRS group, and embryonal histology. Fecal incontinence seems to be a problem.
分析在 CWS-86、-91、-96 和-2002P 试验中接受治疗的会阴/肛周横纹肌肉瘤（PRMS）患者的临床过程、治疗、并发症、结局和生活质量（QOL）。
尽管有多项国际研究试验针对横纹肌肉瘤的治疗，但关于 PRMS 的治疗、结局和 QOL 仅提供了非常有限的信息。
共 35 例 PRMS 患者接受新辅助化疗。局部采用放疗和/或手术治疗，随后进行辅助化疗。通过胃肠道/QOL 调查评估长期功能随访情况。
32 例患者接受了评估（排除 n=3）。8 例患者为胚胎组织学，24 例患者为肺泡组织学。中位年龄为 108 个月（中位随访时间：5.8 年）。5 年总生存率为 47%（95%置信区间：29-64）。16 例 IRS（Intergroup Rhabdomyosarcoma Study）III 和 IV 患者在诊断时存在局部区域淋巴结受累。7 例患者仅接受化疗/手术治疗[5 年无事件生存率（EFS）：85.7%]。11 例患者仅接受放化疗[5 年 EFS：27.3%]。12 例患者接受联合放化疗/手术治疗[5 年 EFS：63.6%]。2 例仅接受化疗的患者死亡。胚胎组织学患者的 5 年 EFS 明显优于肺泡组织学患者（87.5% vs. 39.1%；P=0.013）。部分患者报告有粪便失禁症状。中位 Wexner 粪便失禁评分 9 分（可能范围：0-20），中位 QOL 评分为 90.5 分（适用范围：0-144）。
这些患者的预后仍不理想。有利预后的预测因素包括肿瘤直径小于 5cm、局部区域淋巴结阴性、年龄小于 10 岁、IRS 分组较低和胚胎组织学。粪便失禁似乎是一个问题。
