Maintenance Chemotherapy in Patients With High-Risk Rhabdomyosarcoma: Long-Term Survival Analysis of the European <i>Paediatric</i> Soft Tissue Sarcoma Study Group RMS 2005 Trial
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<sup>2</sup>) once per day on days 1, 8, and 15, plus once daily low-dose cyclophosphamide (25 mg/m<sup>2</sup>; 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 (<i>P</i> = .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; <i>P</i> = .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.
Authors
- Michela Casanova (ORCID: https://orcid.org/0000-0003-0368-7883)
- Johannes H. M. Merks (ORCID: https://orcid.org/0000-0001-7659-1028)
- Raquel Hladun (ORCID: https://orcid.org/0000-0002-3191-1750)
- Rita Alaggio (ORCID: https://orcid.org/0000-0003-3915-3816)
- Maja Česen (ORCID: https://orcid.org/0000-0003-2434-8960)
- Beatrice Coppadoro (ORCID: https://orcid.org/0009-0003-4411-0332)
- Julia Chisholm (ORCID: https://orcid.org/0000-0003-3479-7997)
- Rick Robert van Rijn (ORCID: https://orcid.org/0000-0002-8876-6515)
- Gian Luca De Salvo (ORCID: https://orcid.org/0000-0001-7451-8476)
- Myriam Ben-Arush
- Anne Sophie Defachelles (ORCID: https://orcid.org/0000-0002-4277-9871)
- Florent Guérin (ORCID: https://orcid.org/0000-0002-3487-3745)
- Daniel Orbach (ORCID: https://orcid.org/0000-0002-2520-139X)
- Véronique Minard‐Colin (ORCID: https://orcid.org/0000-0002-0296-5207)
- Gianni Bisogno (ORCID: https://orcid.org/0000-0003-4462-5523)
- Henry Mandeville (ORCID: https://orcid.org/0000-0001-6820-8578)
- Heidi Glosli (ORCID: https://orcid.org/0000-0003-1153-4131)
- Andrea C. Ferrari (ORCID: https://orcid.org/0000-0002-4724-0517)
Institutions
- Universitat Autònoma de Barcelona (ES)
- Oslo University Hospital (NO)
- Royal Marsden NHS Foundation Trust (GB)
- University of Padua (IT)
- Institute of Cancer Research (GB)
- Utrecht University (NL)
- Université Paris Sciences et Lettres (FR)
- Université Paris-Saclay (FR)
- Rambam Health Care Campus (IL)
- Ljubljana University Medical Centre (SI)
- University Medical Center Utrecht (NL)
- Emma Kinderziekenhuis (NL)
- Istituto Oncologico Veneto (IT)
- Royal Marsden Hospital (GB)
- Bambino Gesù Children's Hospital (IT)
- Princess Máxima Center (NL)
- Centre Oscar Lambret (FR)
- Amsterdam University Medical Centers (NL)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Fondazione IRCCS Istituto Nazionale dei Tumori (IT)
- Bicêtre Hospital (FR)
- Institut Curie (FR)
- University of Amsterdam (NL)
Publication Details
- Journal
- Journal of Clinical Oncology
- Published
- 2025-03-07
- DOI
- https://doi.org/10.1200/jco-24-02850
- Citations
- 8
- Primary Topic
- Sarcoma Diagnosis and Treatment
- Type
- article
- Field-Weighted Citation Impact
- 12.15