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Background: In this study, we report the follow‐up results of reduced dose of craniospinal radiotherapy (CSRT) followed by tandem high‐dose chemotherapy (HDCT) in patients with high‐risk medulloblastoma (MB). Methods: Newly diagnosed high‐risk MB patients (metastatic disease, postoperative residual tumor >1.5 cm2, or large cell/anaplastic histology) over 3 years of age were enrolled in this study. Two cycles of pre‐RT chemotherapy, radiotherapy (RT) including reduced‐dose CSRT (23.4 or 30.6 Gy), four cycles of post‐RT chemotherapy, and tandem HDCT were administered. NanoString and DNA sequencing were performed using archival tissues. Results: In all, 40 patients were enrolled, and molecular subgrouping was possible in 21 patients (2 wingless, 3 sonic hedgehog, 8 Group 3, and 8 group 4). All patients including two patients who experienced progression during the induction chemotherapy underwent HDCT. Relapse/progression occurred only in four patients (5‐year cumulative incidence [CI] 10.4 ± 0.3%). However, six patients died from treatment‐related mortality (TRM) (four acute TRMs and two late TRMs) resulting in 18.5 ± 0.5% of 5‐year CI. Taken together, the 5‐year event‐free survival and overall survival were 71.1 ± 8.0% and 73.2 ± 7.9%, respectively. Late effects were evaluated in 25 patients and high‐tone hearing loss, endocrine dysfunction, dyslipidemia, and growth retardation were common. Conclusions: The strategy using tandem HDCT following reduced‐dose CSRT showed promising results in terms of low relapse/progression rate; however, the high TRM rate indicates that modification of HDCT regimen and careful selection of patients who can benefit from HDCT will be needed in the future study. [ABSTRACT FROM AUTHOR]

Abstract: In this study, we evaluated the results of multimodal treatment that included tandem HDCT/auto‐SCT in children with anaplastic ependymomas. Fourteen patients with anaplastic ependymomas were enrolled from 2006 to 2014. Six cycles of induction chemotherapy were administered to all patients before they underwent tandem HDCT/auto‐SCT. Patients who were older than 3 years of age were administered RT after two cycles of induction chemotherapy. In patients under 3 years of age, RT was either omitted or delayed until they reached 3 years of age, if the patients experienced CR after tandem HDCT/auto‐SCT. All patients, including two who experienced disease progression during induction treatment, underwent the first HDCT/auto‐SCT, and 13 subsequently underwent the second HDCT/auto‐SCT. One patient died from hepatic VOD during the second HDCT/auto‐SCT; other toxicities occurring during tandem HDCT/auto‐SCT were manageable. Relapses or progression occurred in seven patients, and five of seven of them remain alive till date after salvage treatment, including surgery and RT. The 5‐year overall and event‐free survival rates were 85.1% ± 9.7% and 50.0% ± 13.4%, respectively. These findings suggest that multimodal treatment including tandem HDCT/auto‐SCT could be a feasible option for improving survival in children with anaplastic ependymomas. [ABSTRACT FROM AUTHOR]

BACKGROUND: In patients with high-risk neuroblastoma, the reduction in primary tumor volume was measured during the early phase of induction chemotherapy as an indicator of early tumor response, and the authors investigated whether the degree of tumor volume reduction could predict outcome in these patients. METHODS: Primary tumor volumes were measured both at diagnosis and at the first tumor response evaluation (after 2 or 3 cycles of induction chemotherapy) in 90 patients with high-risk neuroblastoma who had volumetrically evaluable computed tomography or magnetic resonance scans. If the tumor volume at the first response evaluation was >40% of the initial tumor volume, then the patient was categorized as a poor responder; otherwise, the patient was categorized as a good responder. Outcomes were compared according to the degree of tumor volume reduction at the first response evaluation. RESULTS: The tumor volume reduction was greater in patients who remained relapse free than in patients who had a relapsed tumor (median percentage tumor volume, 21% vs 41.5%; P = .037). The 5-year relapse-free survival rate was higher in the good responders than in the poor responders (83% [95% confidence interval, 72%-94%] vs 51% [95% confidence interval, 31%-71%]; P = .002). In a multivariate analysis of relapse-free survival, a poor early response was identified as an independent, unfavorable prognostic factor (hazard ratio, 4.24; 95% confidence interval, 1.59-11.29; P = .004). CONCLUSIONS: A greater reduction in tumor volume reduction the early phase of induction chemotherapy was associated with a better outcome in patients with high-risk neuroblastoma. Tailoring treatment intensity according to the early tumor response to induction chemotherapy may improve patient outcomes. Cancer 2013. © 2012 American Cancer Society. [ABSTRACT FROM AUTHOR]