Background Survivors of childhood medulloblastoma are particularly susceptible to late effects because of the location of the tumour and the treatment required to achieve a cure. Living with these long-term toxicities is challenging and greater understanding of the impact of the disease and treatment have on health-related quality of life (HRQoL) is needed. Procedure We report a cross-sectional study to assess patient HRQoL outcomes for 52 long-term survivors of medulloblastoma aged 1–25 years of age at diagnosis and treated during a ten-year period at The Royal Marsden Hospital. Child self-reports and parent – proxy reports of PedsQL scores correlate with clinical information, long-term toxicity (assessed using CTCAE) and neurocognitive assessment (using WISC-IV) to examine the impact that disease and treatment have on HRQoL after treatment. Results Reported late toxicities included ataxia (62%), hearing impairment (59%), endocrine disorders (57%) and memory impairment (44%). Reduced HRQoL outcome scores for patients showed a significant correlation with reduced verbal comprehension (0.51; p=0.025), and processing speed (0.5; p=0.04). Memory impairment showed significant association with the cancer module PedsQL (p=0.024) scores. Parents’ perception of their child’s quality of life was lower than the patient’s self-assessment (mean 55.9 for parents and 63.8 for patients, p=0.004). Conclusions The findings from this study confirm the impact of late toxicities and neurocognitive sequelae on HRQoL in patients previously treated for medulloblastoma in childhood and adolescence. In particular verbal comprehension, processing speed and memory impairment influence patient reported outcomes in this cohort.

The International Soft-Tissue Sarcoma Database Consortium (INSTRuCT) consists of a collaboration between the Children’s Oncology Group (COG) Soft Tissue Sarcoma Committee, the European pediatric Soft-Tissue Sarcoma Study Group (EpSSG), and the Cooperative Weichteilsarkom Studiengruppe (CWS). As part of the larger initiative of INSTRuCT to provide consensus expert opinions for clinical treatment of pediatric soft tissue sarcoma, we sought to provide updated, evidenced-based consensus guidelines for local treatment of parameningeal rhabdomyosarcoma using both existing literature as well as recommendations from the relevant cooperative group clinical trials. Overall, parameningeal rhabdomyosarcoma represents a distinctly challenging disease to treat given its location near many critical structures in the head and neck, frequently advanced local presentation, and predilection for local failure. Definitive chemoradiation remains the standard treatment approach for parameningeal rhabdomyosarcoma, with surgery often limited to biopsy or salvage therapy for recurrent disease. In this consensus paper, we specifically discuss consensus guidelines and evidence for definitive local management with radiotherapy, with a focus on imaging for radiotherapy planning, dose and timing of radiation, approach for nodal irradiation, various radiation techniques including proton therapy, and the limited role of surgical resection.

Background: Treatment of children and adolescents with alveolar rhabdomyosarcoma (ARMS) and regional nodal involvement (N1) have been approached differently by North American and European cooperative groups. In order to define the better therapeutic strategy, we analyzed two studies conducted between 2005 and 2016 by the European paediatric Soft tissue sarcoma Study Group (EpSSG) and Children’s Oncology Group (COG). Methods: We retrospectively identified patients with ARMS N1 enrolled in either EpSSG RMS2005 or in COG ARST0531. Chemotherapy in RMS2005 comprised IVADo (ifosfamide, vincristine, dactinomycin, doxorubicin), IVA and maintenance (vinorelbine, cyclophosphamide); in ARST0531 it consisted on either VAC (vincristine, dactinomycin, cyclophosphamide) or VAC alternating with VI (vincristine, irinotecan). Local treatment was similar in both protocols. Results: The analysis of the clinical characteristics of 239 patients showed some differences between study groups: in RMS2005, advanced IRS Group and large tumors predominated. There were no differences in outcomes between the two groups: 5-year event-free survival (EFS), 49% (95%CI=39-59) and 44% (95%CI=30-58), and overall survival (OS), 51% (95%CI=41-61) and 53.6% (95%CI=40-68), in RMS2005 and ARST0531, respectively. In RMS2005, EFS of patients with FOXO1-positive tumors was significantly inferior to those FOXO1-negative (49.3% vs 73%, p=0.034). In contrast, in ARST0531, EFS of patients with FOXO1-positive tumors was 45% compared with 43.8% for those FOXO1-negative. Conclusions: The outcome of patients with ARMS N1 was similar using different schemas of chemotherapy. However, patients with FOXO1 fusion-negative tumors enrolled in RMS2005 showed a significantly better outcome, suggesting that this subgroup may benefit from the EpSSG strategy which included maintenance chemotherapy.