Potential Importance of Early Focal Radiotherapy Following Gross Total Resection for Long-Term Survival in Children With Embryonal Tumors With Multilayered Rosettes

Frontiers in Oncology

Christian Dorfer¹  Thomas Czech¹  Johannes Gojo²  Natalia M. Stepien²  Irene Slavc²  Lisa Mayr²  Andreas Peyrl²  Thomas Pletschko²  Amedeo A. Azizi²  Karin Dieckmann³  Christine Haberler⁴  Sander Lambo⁵  Marcel Kool⁷ 

[1] Department of Neurosurgery, Medical University of Vienna, Vienna, Austria;Department of Pediatrics and Adolescent Medicine and Comprehensive Center for Pediatrics, Medical University of Vienna, Vienna, Austria;Department of Radiotherapy, Medical University of Vienna, Vienna, Austria;Division of Neuropathology and Neurochemistry, Department of Neurology, Medical University of Vienna, Vienna, Austria;Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ) and German Cancer Consortium (DKTK), Heidelberg, Germany;Division of Pediatric Neurooncology, Hopp Children’s Cancer Center Heidelberg (KiTZ), Heidelberg, Germany;Research Department, Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands;

关键词: embryonal tumor with multilayered rosette;    embryonal tumor with abundant neuropil and true rosette;    radiotherapy;    focal radiotherapy;    intrathecal therapy;    embryonal brain tumors;   

DOI  :  10.3389/fonc.2020.584681

来源: DOAJ

【 摘 要 】

Embryonal tumor with multilayered rosettes (ETMR) is a rare, aggressive embryonal central nervous system tumor characterized by LIN28A expression and alterations in the C19MC locus. ETMRs predominantly occur in young children, have a dismal prognosis, and no definitive treatment guidelines have been established. We report on nine consecutive patients and review the role of initiation/timing of radiotherapy on survival. Between 2006 and 2018, nine patients were diagnosed with ETMR. Diagnosis was confirmed histopathologically, immunohistochemically and molecularly. Median age was 25 months (5–38). Location was supratentorial in five, pineal in three, and brainstem in one. Seven patients had a gross total resection, one a partial resection and one a biopsy at initial diagnosis. Chemotherapy augmented with intrathecal therapy started a median of 10 days (7–20) after surgery. Only two patients who after gross total resection received radiotherapy very early on (six weeks after diagnosis) are alive and in complete remission 56 and 50 months after diagnosis. All remaining patients for whom radiotherapy was deferred until the end of chemotherapy recurred, albeit none with leptomeningeal disease. A literature research identified 228 patients with ETMR. Including our patients only 26 (11%) of 237 patients survived >36 months with no evidence of disease at last follow-up. All but two long-term (>36 months) survivors received radiotherapy, ten of whom early on following gross total resection (GTR). GTR followed by early focal radiotherapy and intrathecal therapy to prevent leptomeningeal disease are potentially important to improve survival of ETMR in the absence of effective targeted therapies.

【 授权许可】

Unknown