Clinical study on 48 children with head and neck rhabdomyosarcoma treated by multi-disciplinary therapy

Abstract

Objective To explore the short-term efficacy in children with head and neck rhabdomyosarcoma (HN-RMS) treated by multidisciplinary therapy, and to analyze the prognostic factors, so as to guide the diagnosis and treatment. Methods Patients with HN-RMS admitted at Hematology Oncology Center of Beijing Children′s Hospital (BCH), Capital Medical University between December 2012 and May 2017, were included in this case-observation study.The clinical characteristics were analyzed and the treatment effect and prognostic factors were summarized. Results A total of 48 cases were collected, including 36 boys and 12 girls, with a median age of 4.6 years.Primarysite parameningeal RMS(PM-RMS)(34 cases, 70.8%), orbital(2 cases, 4.2%) and non-orbital, non-parameningeal region(12 cases, 25.0%) were found.Twenty cases belonged to alveolar type(41.7%), and 28 cases were of embryonal type(58.3%). The diameter of the tumor was >5 cm(n=25, 52.1%), and ≤5 cm(n=23, 47.9%). IRS staging: there were 29 cases(60.4%) of stage Ⅱ-Ⅲ, 19 cases(39.6%) of stage Ⅳ; 29 cases(60.4%) of low-medium risk, and 19 cases(39.6%) of high risk.Twenty-three patients(47.9%) received surgery, and 25 cases (52.1%) received biopsy only.All patients (48 cases) received systemic chemotherapy.Twenty patients(41.6%) received external radiation, 15 cases (31.3%)received 125I particle implantation, 6 cases (12.5%)received proton therapy, but 3 cases (6.2%)did not receive radiation.The follow-up time lasted 13-57 months[(24.1±12.3) months]. The 2-year overall survival(OS) rate was (66.4±7.2)%, and 2-year event free survival (EFS) rate was (59.9±7.5)%.Patients with tumor diameter ≤5 cm had higher OS and EFS than patients with tumor diameter >5 cm [2-year OS (87.4±6.8)% vs.(42.9±6.8)%, 2-year EFS (78.8±8.6%) vs.(38.5±10.8)%], and the differences were statistically significant (all P=0.006). Patients with orbital and non-orbital, non-parameningeal RMS had higher OS and EFS than PM-RMS [2-year OS 100% vs.(87.5%±11.7)% vs.(57.0±8.8)%; 2-year EFS 100% vs.(88.9±10.5)% vs.(51.1±8.9)% ], and the differences were statistically significant (P=0.008, P=0.030). Patients who received surgery had higher OS and EFS than those who did not received surgery[2-year OS (80.7±8.8)% vs.(53.3±10.4)%; 2-year EFS (71.1±10.1)% vs.(49.5±10.4)%], and the differences were statistically significant (P=0.008, P=0.026). COX regression analysis showed tumor diameter >5 cm was an adverse prognostic factor (OR=4.124, 95%CI: 1.213-14.025, P=0.023). Conclusions PM-RMS accounted for a high proportion in RMS patients.The primary site and the size of the tumor are the main prognostic factors.Intensive therapy is expected to improve the prognosis of HN-RMS with meningeal invasion. Key words: Head and neck rhabdomyosarcoma; Multi-disciplinary treatment; Parameningeal rhabdomyosarcoma; Child