Outcomes after first relapse of childhood intracranial ependymoma

Abstract

Background: Ependymoma is the third most common malignant CNS tumor in children. Despite multimodal therapy, prognosis of relapsed ependymoma remains poor.Approaches to therapy for relapsed ependymoma are varied. We present a singleinstitution retrospective review of the outcomes after first relapse of intracranial ependymoma in children.Procedure: We performed a retrospective, IRB-approved chart review of patients with recurrent intracranial ependymoma treated at Dana-Farber/Boston Children's Cancer and Blood Disorders Center from 1990 to 2019.Results: Thirty-four patients with relapsed intracranial ependymoma were identified.At initial diagnosis, 11 patients had supratentorial disease, 22 with posterior fossa disease and one with metastatic disease. Median time-to-first relapse was 14.9 months from initial diagnosis (range 1.4-52.5). Seven patients had metastatic disease at first relapse. Gross total resection (GTR) was associated with improved 5-year progressionfree survival (PFS) relative to subtotal resection (STR) and no surgery (p = .005). Localized disease at relapse was associated with improved 5-year overall survival (OS) when compared to metastatic disease (p = .02). Irradiation at first relapse seemed to delay progression but was not associated with statistically prolonged PFS or OS. Tumor location, histology, and chromosomal 1q status did not impact outcome at first relapse, although available molecular data were limited making definitive conclusions difficult.Median time-to-second relapse was 10 months (range 0.7-124). Five-year PFS and OS after first relapse were 19.9% and 45.1%, respectively. Median PFS and OS were 10.0 and 52.5 months after first relapse, respectively. Conclusions:Relapsed intracranial ependymoma has a poor prognosis despite multimodal therapy. Novel therapeutic strategies are desperately needed for this disease.

Toward Improved Diagnosis Accuracy and Treatment of Children, Adolescents, and Young Adults With Ependymoma: The International SIOP Ependymoma II Protocol

“…Patients with metastatic and/or relapsing disease included in the staging phase of the program then in the observational study have a worse prognosis ( 6 , 23 ) and would benefit from the results of the stratum I and stratum II along with BIOMECA analyses. These results combined with other large molecular profiling will help in the design of new dedicated personalized studies.…”

“…Indeed, the recognition that myxopapillary tumors are less indolent than hitherto thought, has led to WHO proposing these are in fact grade II tumors, underlines the importance of including these patients within the umbrella of a clinical trial ( 22 ). In the same way, including patients with metastatic and/or relapsing disease in the staging phase of the program then in the observational study could provide substantial information on the tumor biology and treatment of specific high-risk patients with very poor prognosis ( 16 , 23 ).…”

Molecular Stratification of Childhood Ependymomas as a Basis for Personalized Diagnostics and Treatment

“…Indeed, in DMG, disruption of H3K27me3-mediated epigenetic regulation is associated with an extremely aggressive course of the disease, typically presenting with sustained tumor growth and polychemotherapy resistance [ 56 , 57 , 58 ]. Similarly, effective chemotherapy regimens for PF-EPN-A are missing [ 59 ] and therapeutic options for relapses are extremely limited [ 5 , 60 , 61 , 62 , 63 ].…”

Systemic chemotherapy of pediatric recurrent ependymomas: results from the German HIT-REZ studies

“…While survival-benefits for surgery and radiotherapy have been shown in multiple cohorts of recurrent EPN, results on chemotherapy were less favorable [1,[5][6][7][8]. So far, no uniform chemotherapeutic treatment regimen for recurrent EPN has been established.…”