Outcome of infants and young children with newly diagnosed ependymoma treated on the “Head Start” III prospective clinical trial

Abstract

This study investigates the outcome of children <10 years old with newly-diagnosed ependymoma treated on the prospective multinational “Head Start” III clinical trial. Between April 2004 and July 2009, 19 children with newly-diagnosed ependymoma were enrolled. All children were to receive five induction chemotherapy cycles followed by one consolidation cycle of myelo-ablative chemotherapy and autologous hematopoietic cell rescue. Children between 6 and 10 years of age or with residual tumor prior to consolidation were to receive irradiation thereafter. Median age of 19 children (8 female) was 20 months at diagnosis. Median follow up was 44 months. The primary site was infratentorial in 11 and supratentorial in 8 patients. Gross total resection was achieved in 10 patients. After induction chemotherapy, all three supratentorial ependymoma patients with residual disease achieved a complete response (CR), while only one of six infratentorial patients with residual disease achieved CR. Three infratentorial patients developed progressive disease during induction chemotherapy. All four infratentorial patients with residual disease who underwent autologous hematopoietic cell transplant, failed to achieve CR. Four patients received focal irradiation following chemotherapy. The 3-year event free survival (EFS) and overall survival (OS) for supratentorial ependymoma were 86 ± 13 % and 100 % respectively. The 3-year EFS and OS for infratentorial ependymoma were 27 ± 13 % and 73 ± 13 % respectively. The role of intensive induction and consolidation chemotherapy in deferring irradiation should be investigated further in children with supratentorial ependymoma with residual disease following surgery. This approach appears ineffective in children with infratentorial ependymoma in the absence of irradiation.

Surveillance imaging for high‐grade childhood brain tumors: What to do 10 years after completion of treatment?

“…We found no recurrence of the primary brain tumor, either local or distant, 10 years or more after the end of treatment in the reviewed literature . After combining the latency period from all relapses, median time to relapse was 13.7 months; average minimal latency time was 3.3 months and a maximum of 49.0 months.…”

Progression‐free survival of children with localized ependymoma treated with intensity‐modulated radiation therapy or proton‐beam radiation therapy

“…11,20 It is reasonable to assume that more aggressive tumor resection has been attempted in recent years. Patients in the PRT group were also treated recently after several publications revealed GTR to be the most important prognostic factor.…”

Expression of Methylthioadenosine Phosphorylase (MTAP) in Pilocytic Astrocytomas

“…This is particularly relevant for lymphomas, leukemias [27,29] and nonsmall-cell lung carcinoma [34,45] . Nevertheless, chemotherapy regimens with pemetrexed were not effective for pediatric patients with medulloblastomas, ependymomas and high-grade gliomas [49] , and the addition of methotrexate in the chemotherapeutic treatment of infratentorial ependymomas did not improve the response to these drugs [50] . However, none of these studies assessed MTAP expression in the tumors.…”