Adults can be cured in a high percentage by a multimodal treatment according to pediatric protocols. Toxicity is higher than in children, but acceptable in view of the high remission rate.

Abstract Background Controversy exists as to what may be defined as standard of care (including markers for stratification) for patients with atypical teratoid/rhabdoid tumors (ATRTs). The European Rhabdoid Registry (EU-RHAB) recruits uniformly treated patients and offers standardized genetic and DNA methylation analyses. Methods Clinical, genetic, and treatment data of 143 patients from 13 European countries were analyzed (2009–2017). Therapy consisted of surgery, anthracycline-based induction, and either radiotherapy or high dose chemotherapy following a consensus among European experts. Fluorescence in situ hybridization, multiplex ligation-dependent probe amplification, and sequencing were employed for assessment of somatic and germline mutations in SWItch/sucrose nonfermentable related, matrix associated, actin dependent regulator of chromatin, subfamily B (SMARCB1). Molecular subgroups (ATRT-SHH, ATRT-TYR, and ATRT-MYC) were determined using DNA methylation arrays, resulting in profiles of 84 tumors. Results Median age at diagnosis of 67 girls and 76 boys was 29.5 months. Five-year overall survival (OS) and event-free survival (EFS) were 34.7 ± 4.5% and 30.5 ± 4.2%, respectively. Tumors displayed allelic partial/whole gene deletions (66%; 122/186 alleles) or single nucleotide variants (34%; 64/186 alleles) of SMARCB1. Germline mutations were detected in 26% of ATRTs (30/117). The patient cohort consisted of 47% ATRT-SHH (39/84), 33% ATRT-TYR (28/84), and 20% ATRT-MYC (17/84). Age <1 year, non-TYR signature (ATRT-SHH or -MYC), metastatic or synchronous tumors, germline mutation, incomplete remission, and omission of radiotherapy were negative prognostic factors in univariate analyses (P < 0.05). An adjusted multivariate model identified age <1 year and a non-TYR signature as independent negative predictors of OS: high risk (<1 y + non-TYR; 5-y OS = 0%), intermediate risk (<1 y + ATRT-TYR or ≥1 y + non-TYR; 5-y OS = 32.5 ± 8.7%), and standard risk (≥1 y + ATRT-TYR, 5-y OS = 71.5 ± 12.2%). Conclusions Age and molecular subgroup status are independent risk factors for survival in children with ATRT. Our model warrants validation within future clinical trials.

BACKGROUNDSurgery alone is the appropriate first‐line treatment for patients with mesoblastic nephroma (MN). Nevertheless, there are reports of local recurrences and metastasis, especially in the cellular subtype. The authors evaluated the outcome of patients with MN who were enrolled in either the International Society of Pediatric Oncology (SIOP) 93‐01/GPOH or the SIOP 2001/GPOH Nephroblastoma Study and Trial.METHODSIn total, 50 patients with MN were analyzed. Eleven patients were suspected antenatally of having a renal tumor. The median age at diagnosis was 18.5 days. Central pathologic review was performed for all specimens. The median observation time was 4.2 years.RESULTSForty‐five patients underwent initial surgery. Five patients older than 6 months received preoperative chemotherapy. Twenty‐nine tumors were classic MN, and 21 tumors were cellular MN. Nine patients had a Stage III MN, 5 of those patients had tumor ruptures, and 8 had positive surgical margins. After they underwent nephrectomy, 40 patients received no further treatment. For the entire group, event‐free survival (EFS) (94%) and overall survival (OS) (95%) were excellent. Patients with a cellular MN, patients with age 3 months or older, and patients with Stage III MN had lower EFS. Three patients developed recurrent disease, and 2 of those patients died. Metastases to the brain, lung, and liver were observed in 1 patient.CONCLUSIONSRadical nephrectomy with accurate surgical‐pathologic staging is the standard of care for children with MN. Nonetheless, a subgroup of patients with MN (Stage III cellular MN in patients age 3 months or older) tends to develop recurrences more often. Further prospective studies will be needed to verify this finding and should help determine whether these patients may benefit from adjuvant therapy. Cancer 2006. © 2006 American Cancer Society.

The prognosis of mediastinal teratoma is excellent after complete or microscopically incomplete resection. In children with malignant GCT, the prognosis is favorable with a therapeutic strategy of delayed resection after preoperative chemotherapy. In most children, the diagnosis can be based on elevated tumor markers and imaging. Biopsy is indicated in nonsecreting GCT.