Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study

Bisogno G, Minard-Colin V, Zanetti I, Ferrari A, Gallego S, Davila Fajardo R, Mandeville H, Kelsey A, Alaggio R, Orbach D, Terwisscha van Scheltinga S, Guillen Burrieza G, Ben-Arush M, Glosli H, Mudry P, Ferman S, Devalck C, Defachelles AS, Merks JHM, Jenney M.
J Clin Oncol 2023 May 1;41(13):2342-2349.doi: 10.1200/JCO.22.02093. Epub 2023 Feb 27.

The study was open from October 1st, 2005, to December 31st, 2016 and 1,733 patients were enrolled from 14 countries (Argentina, Belgium, Brazil, Czech Republic, France, Ireland, Israel, Italy, Norway, Switzerland, Slovenia, Spain, The Netherlands, and United Kingdom).
After a median followup of 73.1 months, the 5-year event-free survival (EFS) and overall survival (OS) of the 1,733 patients enrolled were 70.7% and 80.4%, respectively. The results by subgroup: LR (80 patients) EFS 93.7%, OS 96.7%; SR (652 patients) EFS 77.4%, OS 90.6%; HR (851 patients) EFS 67.3%, OS 76.7%; and VHR (150 patients) EFS 48.8%, OS 49.7%. The study demonstrated that around 80% of patients with nonmetastatic RMS are long-term survivors, partly explained by the excellent outcome of LR and SR patients (42.2% of the population), with long-term survival rates above 90%. The LR group results demonstrate that the criteria we used identify a selected group (4.6% of patients) that can be treated with relatively short, low-toxic chemotherapy.
The RMS 2005 trial has modified the existing European standard proposing: (1) a 22-week alkylating-free regimen for LR patients, (2) the reduction of the cumulative dose of ifosfamide in the SR group, (3) the omission of anthracyclines in HR patients (4) 27 weeks of standard chemotherapy (ifosfamide, vincristine and actinomycn D), with the addition of maintenance chemotherapy for HR patients.