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
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.
Article Details
Journal Info
Journal of Clinical Oncology
Lippincott Williams & Wilkins
Authors (18)
Gianni Bisogno
Julia Chisholm
Department of Pediatric Oncology, The Royal Marsden Hospital and The Institute of Cancer Research, Sutton, Surrey, United Kingdom
Raquel Hladun
Pediatric Oncology and Hematology Department, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain
Gian Luca De Salvo
Clinical Research Unit, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy
Florent Guerin
Department of Pediatric Surgery, Bicetre Hospital, Université Paris-Saclay, Le Kremlin Bicetre, France
Michela Casanova
Henry Mandeville
The Royal Marsden Hospital, Sutton, United Kingdom
Rita Alaggio
Pathology Unit, Department of Laboratories, Bambino Gesu Children's Hospital, IRCCS, Rome, Italy
Beatrice Coppadoro
Department of Women's and Children's Health, University of Padua, Padua, Italy
Daniel Orbach
Siredo Oncology Center (Care, Innovation and Research for Children and AYA With Cancer), Institut Curie and University PSL, Paris, France
Andrea Ferrari
Fondazione IRCCS Isttuto Nazionale Tumori, Milano, Italy
Rick van Rijn
Amsterdam UMC, Amsterdam, Netherlands
Anne-Sophie Defachelles
Pediatric and AYA department, Oscar Lambret Center, Lille, France
Myriam Ben-Arush
Joan and Sanford Weill Pediatric Hematology Oncology and Bone Marrow Transplantation Division, Ruth Rappaport Children's Hospital, Rambam Medical Center, Haifa, Israel
Heidi Glosli
Department of Paediatric Research and Department of Paediatric and Adolescent Medicine, Oslo University Hospital, Oslo, Norway
Maja Cesen
University Medical Center, Ljubljana, Slovenia
Johannes H.M. Merks
Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands
Veronique Minard-Colin
Gustave Roussy, Villejuif, France