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Cancer Health Center

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Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Changes to This Summary (08 / 19 / 2014)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

General Information

Recommended Related to Cancer


Note: Separate PDQ summaries on Testicular Cancer Treatment and Levels of Evidence for Cancer Screening and Prevention Studies are also available. Benefits Based on fair evidence, screening for testicular cancer would not result in an appreciable decrease in mortality, in part because therapy at each stage is so effective. Magnitude of Effect: Fair evidence of no reduction in mortality. Study Design: Opinions of respected authorities based on clinical experience, descriptive...

Read the Overview article > >

Added Smith et al. as reference 1.

Revised text to state that between 1975 and 2010, childhood cancer mortality decreased by more than 50%; for rhabdomyosarcoma, the 5-year survival rate increased over the same time, from 53% to 67% for children younger than 15 years and from 30% to 51% for adolescents aged 15 to 19 years.

Added text to state that it is unclear whether response to induction chemotherapy, as judged by anatomic imaging, correlates with the likelihood of survival in patients with rhabdomyosarcoma, because the Intergroup Rhabdomyosarcoma Study Group and Children's Oncology Group studies found no association (cited Rosenberg et al. as reference 36 and level of evidence 3iiDi). However, an Italian study found that patient response did correlate with likelihood of survival.

Cellular Classification

Revised text about alveolar histology to state that about 70% to 80% of alveolar tumors are characterized by translocations between the FOXO1 gene on chromosome 13 and either the PAX3 gene on chromosome 2 or PAX7 gene on chromosome 1. Also added text to state that other rare fusions include PAX-NCOA1 and PAX3-INO80D (cited Shern et al. as reference 14).

Added text to state that in addition to FOXO1 rearrangements, alveolar tumors are characterized by a lower mutational burden than are fusion-negative tumors, with fewer genes having recurring mutations; BCOR and PIK3CA mutations and the amplification of MYCN, MIR17HG, and CDK4 have also been described (cited Chen et al. as reference 22).

Added text to state that embryonal tumors show loss of heterozygosity at 11p15 and gains on chromosome 8; embryonal tumors have a higher background mutation and higher single-nucleotide variant rate than do alveolar tumors, and the number of somatic mutations increases with older age at diagnosis. Also added text to state that genes with recurring mutations include those in the RAS pathway, which together are observed in approximately one-third of cases; other genes with recurring mutations include FGFR4, PIK3CA, CTNNB1, FBXW7, and BCOR, all of which are present in fewer than 10% of cases.

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