Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.The RMS2005 study included two phase III randomized trials for high-risk (HR) and observational trials for low (LR), standard (SR), and very high-risk (VHR) patients who have been partially reported. Herein, we present a comprehensive report of results achieved for the complete unselected nonmetastatic cohort and analyze the evolution of treatment in comparison with previous European protocols. After a median follow-up of 73.1 months, the 5-year event-free survival (EFS) and overall survival (OS) of the 1,733 patients enrolled were 70.7% (95% CI, 68.5 to 72.8) and 80.4% (95% CI, 78.4 to 82.3), respectively. The results by subgroup: LR (80 patients) EFS 93.7% (95% CI, 85.5 to 97.3), OS 96.7% (95% CI, 87.2 to 99.2); SR (652 patients) EFS 77.4% (95% CI, 73.9 to 80.5), OS 90.6% (95% CI, 87.9 to 92.7); HR (851 patients) EFS 67.3% (95% CI, 64.0 to 70.4), OS 76.7% (95% CI, 73.6 to 79.4); and VHR (150 patients) EFS 48.8% (95% CI, 40.4 to 56.7), OS 49.7% (95% CI, 40.8 to 57.9). The RMS2005 study demonstrated that 80% of children with localized rhabdomyosarcoma could be long-term survivors. The study has established the standard of care across the European pediatric Soft tissue sarcoma Study Group countries with the confirmation of a 22-week vincristine/actinomycin D regimen for LR patients, the reduction of the cumulative ifosfamide dose in the SR group, and for HR disease, the omission of doxorubicin and the addition of maintenance chemotherapy.

Centre for Rare Disorders Division of Paediatric and Adolescent Medicine Oslo University Hospital Oslo Norway

Department of Paediatric Histopathology Manchester University Foundation Trust Manchester United Kingdom

Department of Paediatric Oncology Children's Hospital for Wales Heath Park Cardiff United Kingdom

Department of Pediatric and Adolescent Oncology Gustave Roussy Université Paris Saclay Villejuif France

Department of Pediatric Oncology and Hematology Hospital Universitari Vall d'Hebron Barcelona Spain

Department of Pediatric Surgery Hospital Universitari Vall d'Hebron Barcelona Spain

Department of Radiotherapy The Royal Marsden Hospital and The Institute of Cancer Research Sutton Surrey United Kingdom

Division of Imaging and Oncology University Medical Center Utrecht Utrecht University Utrecht the Netherlands

Pathology Unit Department of Laboratories Bambino Gesu Children's Hospital IRCCS Rome Italy

Pediatric and AYA Oncology Unit Oscar Lambret Center Lille France

Pediatric Hematology and Oncology Hôpital Universitaire des Enfants Reine Fabiola HUDERF ULB HUB Brussels Belgium

Pediatric Hematology Oncology Division University Hospital of Padua Padua Italy

Pediatric Oncology Department National Cancer Institute Rio de Janeiro Brazil

Pediatric Oncology Department University Hospital Brno and Faculty of Medicine Masaryk University Brno Czech Republic

Pediatric Oncology Unit Fondazione IRCCS Istituto Nazionale Tumori Milano Italy

Princess Máxima Center for Pediatric Oncology Utrecht the Netherlands

Radiation Oncology University Medical Center Utrecht Utrecht the Netherlands

Rambam Medical Center Joan and Sanford Weill Pediatric Hematology Oncology and Bone Marrow Transplantation Division Ruth Rappaport Children's Hospital Haifa Israel

SIREDO Oncology Center Institut Curie PSL University Paris France

Citace poskytuje Crossref.org

Zobrazit více v PubMed

ClinicalTrials.gov
NCT00339118

EudraCT
2005-000217-35