Objective: To develop a reasonable protocol for childhood rhabdomyosarcoma (RS) to improve the prognosis of this disease.
Methods: The protocol RS-99, developed on the base of pathological examination, clinical staging and grouping was used for 30 RS patients, 15 male and 15 female, aged 53 months (15-180 months). For the low-risk patients the regimen VCP and IeV were used alternately for 6 treatment courses and then local radiotherapy was given, for the median and high risk patients, regimen AVCP and IEV were used alternately for 6 courses, local radiotherapy and selective operation were performed, and then DEV and IeV were used alternately for 6 courses, and for the high-risk patients DEV and IeV were used alternately for 18 courses in total and then hematopoietic stem cell transplantation was conducted.
Results: Twenty-three of the 30 patients reached complete response (CR) 17 of which remained in CR for 37 (32-92) months, and 7 of the 30 patients achieved partial response (PR) but their condition worsened later. Relapse was seen in 6 patients 15 (7-38) months later. The 5 year event-free survival (EFS) rate was 47% and the overall survival (OS) rate was 52%. All the 10 stage I and II patients, 5 of the 8 stage III patients, and only 2 of the 12 stage IV patients still remained in CR.
Conclusion: The protocol RS-99 is effective on the RS at stages I, II, and III, however, is ineffective on the disease at stage IV.