Although patients with recurrent or progressive rhabdomyosarcoma sometimes achieve complete remission with secondary therapy, the long-term prognosis is usually poor.[1,2] The prognosis is most favorable (5-year survival rates, 50%-70%) for children who initially present with Stage 1 or Group I disease and embryonal histology and who have small tumors, and for those who have a local or regional nodal recurrence.[1,2,3] A retrospective analysis of children with recurrence after initial presentation with localized rhabdomyosarcoma of the orbit reported 80% survival 5 years after recurrence with aggressive retrieval therapy.[Level of evidence: 3iiA] The small number of children with botryoid histology who relapse have a similarly favorable prognosis. Most other children who relapse have an extremely poor prognosis. A retrospective review of rhabdomyosarcoma patients from German soft tissue sarcoma trials identified time to recurrence as an important independent prognostic factor. Shorter time to recurrence was associated with higher risk of mortality from recurrent rhabdomyosarcoma.[Level of evidence: 3iiB] European investigators performed a retrospective review of patients with rhabdomyosarcoma enrolled on cooperative group trials who experienced recurrence. They identified metastatic (as opposed to local) recurrence, prior radiation therapy, initial tumor size (>5 cm), and time to relapse (<18 months) as unfavorable prognostic features for survival after recurrence. In a retrospective review from the German Cooperative Soft Tissue Sarcoma Group, patients with alveolar rhabdomyosarcoma who relapsed with a single-disease focus and who received subsequent multiagent chemotherapy plus adequate local-relapse therapy (complete resection or gross resection with radiation therapy) had a better probability of long-term disease control than did patients with disseminated recurrences and/or tumors treated without adequate local-relapse therapy.[Level of evidence: 3iiA]
The selection of further treatment depends on many factors, including the site(s) of recurrence, previous treatment, and individual patient considerations. Treatment for local or regional recurrence may include wide local excision or aggressive surgical removal of tumor, particularly in the absence of widespread bony metastases.[8,9] Some survivors have also been reported after surgical removal of only one or a few metastases in the lung. Radiation therapy should be considered for patients who have not already received radiation therapy in the area of recurrence, or rarely for those who have received radiation therapy but for whom surgical excision is not possible. Previously unused, active, single agents or combinations of drugs may also enhance the likelihood of disease control.
Many of the medical and scientific terms used in this summary are found in the NCI Dictionary of Genetics Terms. When a linked term is clicked, the definition will appear in a separate window.
Creating evidence-based summaries on cancer genetics is challenging because the rapid evolution of new information often results in evidence that is incomplete or of limited quality. In addition, established methods for evaluating the quality of the evidence are available for some, but not all, aspects of...