Maintenance Chemotherapy in Patients With High-Risk Rhabdomyosarcoma: Long-Term Survival Analysis of the European Paediatric Soft Tissue Sarcoma Study Group RMS 2005 Trial
Year: 2025
Type: article
Study design: Clinical Trial
Abstract: The European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) RMS 2005 trial evaluated maintenance chemotherapy in high-risk rhabdomyosarcoma (RMS). Patients were randomly assigned to either discontinue treatment (standard arm) or receive six 28-day cycles of vinorelbine (25 mg/m 2 ) once per day on days 1, 8, and 15, plus once daily low-dose cyclophosphamide (25 mg/m 2 ; experimental arm). Initial results showed improved overall survival (OS), but disease-free survival (DFS) improvement was not statistically significant. This report presents mature survival outcomes after extended follow-up. Between April 2006 and December 2016, 186 patients were enrolled in the standard arm and 185 in the experimental arm. After a median follow-up of 122.1 months from diagnosis and 114 months from random assignment, recurrence, progression, or death occurred in 103 patients (61 standard arm, 42 experimental arm). The 10-year DFS was 66.5% (95% CI, 59 to 74) in the standard arm versus 77.1% (95% CI, 70.3 to 82.5) in the experimental arm ( P = .025). Corresponding 10-year OS rates were 70.8% (95% CI, 63.3 to 77.0) and 82.9% (95% CI, 76.6 to 87.7; P = .0099). Long-term results of the RMS2005 trial confirm the survival benefit of maintenance chemotherapy with vinorelbine and low-dose cyclophosphamide for patients with high-risk RMS.
Source: Journal of Clinical Oncology
DOI: 10.1200/jco-24-02850
Authors: Gianni Bisogno, Julia Chisholm, Raquel Hladun, Gian Luca De Salvo, Florent Guerin, Michela Casanova, Henry Mandeville, Rita Alaggio, Beatrice Coppadoro, Daniel Orbach, Andrea Ferrari, Rick van Rijn, Anne-Sophie Defachelles, Myriam Ben-Arush, Heidi Glosli, Maja Cesen, Johannes H.M. Merks, Véronique Minard-Colin
Institutions: University of Padua, Azienda Ospedale - Università Padova, Institute of Cancer Research, Royal Marsden Hospital, Universitat Autònoma de Barcelona, Vall d'Hebron Hospital Universitari, Istituto Oncologico Veneto, Université Paris-Saclay, Bicêtre Hospital, Fondazione IRCCS Istituto Nazionale dei Tumori, Bambino Gesù Children's Hospital, Université Paris Sciences et Lettres, Institut Curie, Emma Kinderziekenhuis, Amsterdam University Medical Centers, University of Amsterdam, Centre Oscar Lambret, Rambam Health Care Campus, Oslo University Hospital, Ljubljana University Medical Centre, Utrecht University, University Medical Center Utrecht, Princess Máxima Center
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: green
Funders: Bristol-Myers Squibb, Pfizer, Genentech
OpenAlex ID: W4408214719