Prognostic factors in metastatic rhabdomyosarcomas: results of a pooled analysis from United States and European cooperative groups.
OBJECTIVE
To identify risk factors associated with outcome in children with metastatic rhabdomyosarcoma in a large cohort of patients


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).


CONCLUSIONS
This analysis identified subsets of patients with metastatic rhabdomyosarcoma with different outcomes to current therapy and offers a strategy to define patient candidates for experimental approaches to treatment.
Oberlin
Odile
O
Department of Paediatric Oncology, Institut Gustave-Roussy, Rue Camille Desmoulins, 94805 Villejuif Cedex, France. oberlin@igr.fr
Rey
Annie
A
Lyden
Elizabeth
E
Bisogno
Gianni
G
Stevens
Michael C G
MC
Meyer
William H
WH
Carli
Modesto
M
Anderson
James R
JR
D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D019046:Bone Marrow Neoplasms / Q000556:secondary; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008175:Lung Neoplasms / Q000556:secondary; D008297:Male; D012208:Rhabdomyosarcoma / Q000188:drug therapy* / Q000473:pathology / Q000556:secondary; D012307:Risk Factors; D015996:Survival Rate; D016896:Treatment Outcome