Treatment for soft tissue sarcoma in childhood and adolescence. Austrian results within the CWS 96 study.
ABSTRACT The aim of the CWS 96 Study was to achieve an optimal treatment in children and adolescents with soft tissue sarcoma (STS) implementing a further refinement of risk-adapted allocation to chemotherapy, surgery and radiotherapy.
Treatment stratification was based on tumour histology, TNM status, postsurgical stage, localisation and age. Local tumour control was ensured by surgery and risk-adapted radiotherapy.
From 1995 to 2002, 89 patients were registered in Austria. The 3-year event-free survival (EFS) and overall survival rates (OS) were 63% +/- 6% and 71% +/- 6%, respectively. 59/89 patients had localised RMS-like (rhabdomayosarcoma) STS (EFS 73% +/- 7%), 14 had localised NON-RMS STS (EFS 54% +/- 16%) and 15 patients had metastatic disease at diagnosis (EFS 33% +/- 12%), 1 patient had fibromatosis. The EFS rates at 3 years in patients with localised RMS-like tumours according to risk group were 92% +/- 8% for low and standard risk (12 patients) and 67% +/- 8% for high risk (47 patients). Favourable primary tumour sites of nonmetastatic RMS-like STS i.e. orbit, head/neck nonparameningeal or genitourinary non-bladder/prostate were diagnosed in 15 patients (1/15 patients died). In 44 patients with unfavourable localisation such as parameningeal, genitourinary bladder/prostate, extremity and others, 7 deceased. The 3 year EFS according to histology in patients with RMS-like STS was 61% +/- 11% for RME (embryonal RMS ) (28 patients) and 71% +/- 15% for RMA (alveolar RMS) (10 patients). The most common treatment failure was local relapse occurring in 21% of patients in the high-risk group.
Risk-adapted individualisation of treatment led to a reduction of chemotherapy in the low and standard risk group without compromising survival. The outcome of RME and RMA was similar in this cohort of patients. These preliminary results after a median observation time of 2.5 years confirm the CWS 96 strategy.
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ABSTRACT: Purpose Children suffering from paratesticular rhabdomyosarcoma (RMS) have a favorable prognosis. Surgical treatment problems are inadequate primary transscrotal approaches, incomplete tumor resections, and the necessity of secondary hemiscrotectomy. The aim of this study was to evaluate the necessity of hemiscrotectomy regarding local relapse and outcome. Materials and Methods One-hundred-seventy-three patients with diagnosis of paratesticular RMS were enrolled into the trials between 1986 and 2008. Seventeen patients were excluded due to an incomplete data set and alveolar histology. One-hundred-fifty six patients with embryonal subtype (RME) were finally analyzed. All patients were treated according to the treatment protocols of the Cooperative Soft Tissue Sarcoma (CWS) trials including multiagent chemotherapy, tumor resection and / or radiotherapy. Results The five year overall survival rate was 91.5% ± 2.4 for all RME patients. Twenty-eight patients underwent transscrotal approaches initially. 12/28 RME patients were treated with hemiscrotectomy (5-year-EFS 91.7%±8) and 16/28 patients without hemiscrotectomy (5-year-EFS 93.8%±6.1). Additionally, 13/156 patients underwent an inguinal approach and hemiscrotectomy due to suspicious tumor infiltration of the scrotal skin (5-year-EFS was 84.6%±10). Relapses were observed in 3/12 patients after transscrotal approach and hemiscrotectomy (one locoregional lymph node and two metastatic). One metastatic relapse was observed in the patients treated with a transscrotal approach without hemiscrotectomy. 1/13 patients with inguinal approach and hemiscrotectomy had locoregional relapse and died of disease. Conclusions Hemiscrotectomy seems not to be mandatory in patients after transscrotal approaches regarding outcome and local relapse. Nevertheless, hemiscrotectomy should be probably performed if the scrotal skin is infiltrated.The Journal of urology 09/2014; 192(3). DOI:10.1016/j.juro.2014.03.005 · 3.75 Impact Factor
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ABSTRACT: Alveolar Rhabdomyosarcomas (RMA) are characterized by chromosomal translocations, fusing the PAX3 or PAX7 gene with FKHR in about 85%. Previous studies have suggested that the fusion type is associated with prognosis. In order to investigate the predictive value of the PAX-FKHR fusion status on disease outcome of patients with RMA treated in the CWS trials we performed a retrospective analysis. Between 1986 and 2004, out of 446 patients with RMA treated in four consecutive CWS trials, tumor samples from 126 patients were available for RT-PCR analysis. Survival depending on fusion status in context with known clinical risk-factors was analyzed. Out of 126 samples, 121 had adequate quality for PAX-FKHR fusion status analysis. PAX-FKHR fusions were detected in 101 samples: 60% PAX3-FKHR and 24% PAX7-FKHR fusions, 17% were fusion-negative. There was no significant difference in survival between patients with PAX3-FKHR versus PAX7-FKHR positive tumors. The fusion transcript negative cohort showed a more favorable outcome than the fusion transcript positive cohort among patients with metastatic disease. From the established clinical risk-factors none was associated with a significantly higher risk of failure or death in a multivariate analysis. PAX-FKHR fusion type was not a significant predictor for survival in our analysis. More extensive molecular analyses are needed to identify features with prognostic relevance and useful therapeutic impact.Pediatric Blood & Cancer 09/2011; 57(3):406-14. DOI:10.1002/pbc.22958 · 2.56 Impact Factor
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ABSTRACT: BACKGROUND: Recurrences in primary localized alveolar rhabdomyosarcoma (RMA) are common. Post-relapse survival is poor. We evaluated prognostic factors including relapse treatment in patients with recurrent RMA. METHODS: Relapses occurred in 115/235 patients with nonmetastatic RMA treated in four consecutive CWS-trials after achievement of a complete remission. Sufficient information about post-relapse treatment and outcome could be obtained in 99 patients and was retrospectively analyzed. RESULTS: Nine of 99 patients received no salvage therapy and died after a median of 2 months. The remaining 90 patients received multimodal relapse treatment including mandatory chemotherapy. Recurrences were grossly resected in 39 patients; 57 patients received radiation. At a median follow-up from relapse of 8 years, 20 patients were alive and disease-free (5-year post-relapse survival [PROS] 21.3 ± 8). All surviving patients apart from a single individual had an isolated, circumscribed recurrence. Sixteen of 20 survivors were treated with adequate local relapse therapy (ALRT, i.e., either complete resection or gross resection + radiation). Survival in the subgroup of 27 individuals with circumscribed recurrences and ALRT was significantly better (PROS 53.7 ± 19) compared with disseminated recurrences and/or tumors treated without ALRT. Absence of primary lymph node involvement, circumscribed relapses, ALRT, and achievement of a second CR were identified as independent favorable risk factors. CONCLUSION: Post-relapse survival for primary localized RMA is generally poor. However, certain patient groups differed significantly in their likelihood of survival and 50% of patients with circumscribed relapses treated with ALRT survived. These findings may form the basis for an evidence-based risk-stratification for recurrent disease including relapse treatment. Pediatr Blood Cancer © 2013 Wiley Periodicals, Inc.Pediatric Blood & Cancer 08/2013; 60(8). DOI:10.1002/pbc.24488 · 2.56 Impact Factor