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Prognostic Factors in Metastatic Rhabdomyosarcomas: Results of a Pooled Analysis From United States and European Cooperative Groups

Odile Oberlin, Annie Rey, Elizabeth Lyden, Gianni Bisogno, Michael C.G. Stevens, William H. Meyer, Modesto Carli, James R. Anderson

From the Departments of Paediatrics and Biostatistics, Institut Gustave Roussy, Villejuif, France; Department of Preventive and Societal Medicine, University of Nebraska Medical Center, Omaha, NE; Department of Pediatrics, Hematology/Oncology Division, University Hospital of Padova, Padova, Italy; Institute of Child Life and Health, University of Bristol, Bristol, United Kingdom; Department of Pediatrics, Hematology/Oncology Section, University of Oklahoma Health Science Center, Oklahoma City, OK

Corresponding author: Odile Oberlin, MD, Department of Paediatric Oncology, Institut Gustave-Roussy, Rue Camille Desmoulins, 94805 Villejuif Cedex, France; e-mail: oberlin{at}igr.fr

Purpose: To identify risk factors associated with outcome in children with metastatic rhabdomyosarcoma in a large cohort of patients

Patients and Methods: Pooled data were obtained from 788 patients treated in nine studies performed by European and American cooperative groups. Clinical factors, including age, histology, site of primary, and site(s) and number of sites of metastatic disease, were correlated with event-free survival (EFS) and overall survival (OS).

Results: Seven hundred eighty-eight patients were eligible for analysis. The 3-year OS and EFS were 34% (SE, 1.7) and 27% (SE, 1.6), respectively. By univariate analysis, 3-year EFS was significantly and adversely influenced by age, alveolar histology, location of primary tumor in unfavorable site (defined as extremity and "other" sites), presence of three or more sites of metastatic disease, and the presence of bone or bone marrow involvement. By multivariate analysis, EFS was strongly correlated to all factors except histology. Relative risks were 1.6 for age younger than 1 year or at least 10 years, 1.4 for unfavorable site of primary tumor, 1.4 for bone or bone marrow involvement, 1.4 for three or more metastatic sites. EFS was 50% for patients without any of these four adverse factors and was respectively 42%, 18%, 12%, and 5% in patients with one, two, three, or four factors (P < .0001).

Conclusion: This analysis identified subsets of patients with metastatic rhabdomyosaroma with different outcomes to current therapy and offers a strategy to define patient candidates for experimental approaches to treatment.

Presented in poster form at the 43rd Annual Meeting of the American Society of Clinical Oncology, June 1-5, 2007, Chicago, IL.