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Childhood Rhabdomyosarcoma Treatment (PDQ®)

Health Professional Version
Last Modified: 08/19/2014

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

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.

Stage Information

Added text about a retrospective study of 1,687 children with rhabdomyosarcoma enrolled in Intergroup studies from 1991 to 2004 that suggests that about one-third of patients can have limited staging procedures that eliminate bone marrow and bone scan examinations at diagnosis (cited Weiss et al. as reference 5).

Previously Untreated Childhood Rhabdomyosarcoma

Revised text to state that in a retrospective trial, starting radiation therapy (RT) within 2 weeks of diagnosis for patients with signs of meningeal impingement was associated with lower rates of local failure but was of borderline significance. Also added text to state that a subsequent comparison of local control, failure-free survival, and overall survival rates showed no statistical difference between early irradiation (day 0) for Group III patients in IRS-IV with cranial nerve palsy and/or cranial base erosion versus later initiation of RT (week 12) for Group III patients in D9603 with similar evidence of meningeal involvement, suggesting that early RT for this group of patients is not necessary. However, both studies administered early irradiation to all patients with intracranial extension of the primary tumor (cited Spalding et al. as reference 44 and level of evidence 2A).

Added text to state that an alternative strategy, used in European International Society of Pediatric Oncology protocols, has been to avoid major radical surgery when possible and omit external-beam RT if complete disappearance of tumor can be achieved by chemotherapy and conservative surgical procedures. The goal is to preserve a functional bladder and prostate without incurring the late effects of RT or having to perform a total cystectomy/prostatectomy. From 1984 to 2003, 172 patients with nonmetastatic bladder and/or bladder/prostate rhabdomyosarcoma were accrued, and the 5-year overall survival rate was 77%. Fifty percent of the 119 survivors had no significant local therapy, and only 26% received RT (cited Jenney et al. as reference 93 and level of evidence 3iiA).

Added Kirsch et al. as reference 99 and level of evidence 3iA.

Added text to state that high-dose chemotherapy with autologous and allogeneic stem cell rescue has been evaluated in a limited number of patients with rhabdomyosarcoma. The use of this modality has failed to improve the outcomes of patients with newly diagnosed or recurrent rhabdomyosarcoma (cited Thiel et al. as reference 132).

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ NCI's Comprehensive Cancer Database pages.