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Newly Diagnosed Metastatic Intracranial Ependymoma in Children: Frequency, Molecular Characteristics, Treatment, and Outcome in the Prospective HIT Series.

ABSTRACT: BACKGROUND:Data on frequency, clinical presentation, and outcome of primary metastatic intracranial ependymoma in children are scarce. PATIENTS AND METHODS:Prospective data on patients younger than 21?years with metastatic intracranial ependymoma at first diagnosis, registered from 2001 to 2014 in the HIT-2000 trial and the HIT-2000 Interim Registry, were analyzed. RESULTS:Of 453 registered patients with intracranial ependymoma and central neuropathology review, initial staging included spinal magnetic resonance imaging in all patients and lumbar cerebrospinal fluid (CSF) analysis in 402 patients. Ten patients (2.2%) had metastatic disease, including three with microscopic CSF positivity only (M1 metastasis stage, 0.7% of patients with CSF staging). Location of the primary tumor was supratentorial in four patients (all supratentorial RELA-fused ependymoma [ST-EPN-RELA]) and within the posterior fossa in five patients (posterior fossa ependymoma type A [PF-EPN-A], n?=?4; posterior fossa ependymoma not further classifiable, n?=?1), and multifocal in one patient.All four patients with ST-EPN-RELA were alive in first or second complete remission (CR) 7.5-12.3?years after diagnosis. All four patients with macroscopic metastases of posterior fossa or multifocal ependymoma died. Three patients with initial M1 stage (ST-EPN-RELA, n?=?1; PF-EPN-A, n?=?2) received chemotherapy and local irradiation and were alive in second or third CR 3.0-9.7?years after diagnosis. Progression-free and overall survival of the entire cohort at 5?years was 13% (±6%), and 58% (±16%), respectively. CONCLUSION:Primary metastatic disease is rare in children with intracranial ependymoma. Prognosis may depend on molecular subgroup and extent of dissemination, and relevance of CSF analysis for initial staging remains to be clarified. IMPLICATIONS FOR PRACTICE:Childhood ependymoma presenting with metastasis at first diagnosis is very rare with a frequency of 2.4% in this population-based, well-characterized cohort. Detection of microscopic metastases in the cerebrospinal fluid was extremely rare, and impact on prognosis and respective treatment decision on irradiation field remains unclear. Initial metastatic presentation occurs in both supratentorial RELA-fused ependymoma and posterior fossa ependymoma. Prognosis may differ according to extent of metastasis and biological subgroup, with poor prognosis in diffusely spread metastatic posterior fossa ependymoma even after combination therapy with both intensive chemotherapy and craniospinal irradiation, which may help to guide individual therapeutic decisions for future patients.

SUBMITTER: Benesch M

PROVIDER: S-EPMC6738295 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature

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Newly Diagnosed Metastatic Intracranial Ependymoma in Children: Frequency, Molecular Characteristics, Treatment, and Outcome in the Prospective HIT Series.

Benesch Martin M Mynarek Martin M Witt Hendrik H Warmuth-Metz Monika M Pietsch Torsten T Bison Brigitte B Pfister Stefan M SM Pajtler Kristian W KW Kool Marcel M Schüller Ulrich U Pietschmann Klaus K Juhnke Björn-Ole BO Tippelt Stephan S Fleischhack Gudrun G Schmid Irene I Kramm Christof M CM Vorwerk Peter P Beilken Andreas A Classen Carl Friedrich CF Hernáiz Driever Pablo P Kropshofer Gabriele G Imschweiler Thomas T Lemmer Andreas A Kortmann Rolf-Dieter RD Rutkowski Stefan S von Hoff Katja K

The oncologist 20190308 9

<h4>Background</h4>Data on frequency, clinical presentation, and outcome of primary metastatic intracranial ependymoma in children are scarce.<h4>Patients and methods</h4>Prospective data on patients younger than 21 years with metastatic intracranial ependymoma at first diagnosis, registered from 2001 to 2014 in the HIT-2000 trial and the HIT-2000 Interim Registry, were analyzed.<h4>Results</h4>Of 453 registered patients with intracranial ependymoma and central neuropathology review, initial sta ...[more]

PMID: 30850560

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Project description:Purpose: The purpose of this study was to analyze the frequency and prognosis of pulmonary metastases in newly diagnosed gastric cancer using population-based data from SEER. Methods: Patients with gastric cancer and pulmonary metastases (GCPM) at the time of diagnosis in advanced gastric cancer were identified using the Surveillance, Epidemiology and End Result (SEER) database of the National Cancer Institute from 2010 to 2014. Multivariable logistic regression was performed to identify predictors of the presence of GCPM at diagnosis. Receiver operator characteristics analysis was performed to significant predictors on multivariable logistic regression and was then assessed with Delong's test. Multivariable Cox regression was developed to identify factors associated with all-cause mortality and gastric cancer-specific mortality. Survival curves were obtained according to the Kaplan-Meier method and compared using the log-rank test. Results: We identified 1,104 patients with gastric cancer and pulmonary metastases at the time of diagnosis, representing 6.02% of the entire cohort and 15.19% of the subset with metastatic disease to any distant site. Among the entire cohort, multivariable logistic regression identified six factors (younger, upper 1/3 of stomach, intestinal-type, T4 staging, N1 staging, and presence of more extrapulmonary metastases to liver, bone, and brain) as positive predictors of the presence of pulmonary metastases at diagnosis. The value of AUC for the multivariable logistic regression model was 0.775. Median survival among the entire cohort with GCPM was 3.0 months (interquartile range: 1.0-9.0 mo). Multivariable Cox model in SEER cohort confirmed five factors (diagnosis at previous period, black race, adverse pathology grade, absence of chemotherapy, and presence of more extrapulmonary metastases to liver, bone, and brain) as negative predictors for overall survival. Conclusions: The findings of this study provided population-based estimates of the frequency and prognosis for GCPM at time of diagnosis. The multivariable logistic regression model had an acceptable performance to predict the presence of PM. These findings may provide preventive guidelines for the screening and treatment of PM in GC patients. Patients with high risk factors should be paid more attention before and after diagnosis.

Project description:BACKGROUND:This report documents the clinical characteristics, molecular grouping, and outcome of young children with ependymoma treated prospectively on a clinical trial. METHODS:Fifty-four children (aged ?3 y) with newly diagnosed ependymoma were treated on the St Jude Young Children 07 (SJYC07) trial with maximal safe surgical resection, 4 cycles of systemic chemotherapy, consolidation therapy using focal conformal radiation therapy (RT) (5-mm clinical target volume), and 6 months of oral maintenance chemotherapy. Molecular groups were determined by tumor DNA methylation using Infinium Methylation EPIC BeadChip and profiled on the German Cancer Research Center/Molecular Neuropathology 2.0 classifier. RESULTS:One of the 54 study patients had metastases (cerebrospinal fluid positive) at diagnosis. Gross or near-total resection was achieved in 48 (89%) patients prior to RT. At a median follow-up of 4.4 years (range, 0.2-10.3 y), 4-year progression-free survival (PFS) was 75.1% ± 7.2%, and overall survival was 92.6% ± 4.4%. The molecular groups showed no significant difference in PFS (4-year estimates: posterior fossa ependymoma group A [PF-EPN-A; 42/54], 71.2% ± 8.3%; supratentorial ependymoma positive for v-rel avian reticuloendotheliosis viral oncogene homolog A [ST-EPN-RELA; 8/54], 83.3% ± 17.0%; and supratentorial ependymoma positive for Yes-associated protein [4/54], 100%, P = 0.22). Subtotal resection prior to RT was associated with an inferior PFS compared with gross or near-total resection (4-year PFS: 41.7% ± 22.5% vs 79.0% ± 7.1%, P = 0.024), as was PF-EPN-A group with 1q gain (P = 0.05). Histopathologic grading was not associated with outcomes (classic vs anaplastic; P = 0.89). CONCLUSIONS:In this prospectively treated cohort of young children with ependymoma, ST-EPN-RELA tumors had a more favorable outcome than reported from retrospective data. Histologic grade did not impact outcome. PF-EPN-A with 1q gain and subtotal resection were associated with inferior outcomes.

Dataset Information

Newly Diagnosed Metastatic Intracranial Ependymoma in Children: Frequency, Molecular Characteristics, Treatment, and Outcome in the Prospective HIT Series.

Publications

Newly Diagnosed Metastatic Intracranial Ependymoma in Children: Frequency, Molecular Characteristics, Treatment, and Outcome in the Prospective HIT Series.

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