Details

Autor(en) / Beteiligte

• Khan, Sara
• Solano-Paez, Palma
• Suwal, Tannu
• Al-Karmi, Salma
• Lu, Mei
• Ho, Ben
• Fouladi, Maryam
• Leary, Sarah
• Levy, Jean M Mulcahy
• Lassaletta, Alvaro
• Rivas, Eloy
• Reddy, Alyssa
• Gillespie, G Yancey
• Gupta, Nalin
• Yalon-Oren, Michal
• Amariglio, Laura
• Nakamura, Hideo
• Wu, Kuo-Sheng
• Wong, Tai-Tong
• Ra, Young-Shin
• Spina, Milena La
• Emanuele, Policlinico Vittorio
• Massimi, Luca
• Buccoliero, Anna Maria
• Hansford, Jordan R
• Grundy, Richard G
• Adamek, Dariusz
• Fangusaro, Jason
• Scharnhorst, David
• Johnston, Donna
• Lafay-Cousin, Lucie
• Camelo-Piragua, Sandra
• Kabbara, Nabil
• Gajjar, Amar
• Boutarbouch, Mahjouba
• da Costa, Maria Joao Gil
• Hanson, Derek
• Wood, Paul
• Al-Hussaini, Maysa
• Amayiri, Nisreen
• Wang, Yin
• Catchpoole, Daniel
• Michaud, Jean
• Bendel, Anne E
• Ellezam, Benjamin
• Gerber, Nicholas
• Plant, Ashley
• Jeffery, Rubens
• Dunham, Christopher
• Moertel, Christopher
• Walter, Andrew
• Ziegler, David
• Dodgshun, Andrew
• Gottardo, Nicholas
• Demir, Ahmet
• Ramanujachar, Ramya
• Raabe, Eric
• Mary, Shago
• Dirks, Peter
• Taylor, Michael
• Eugene, Hwang
• Lindsey, Holly
• Tihan, Tarik
• Mette, Jorgensen
• Dahl, Christine
• Low, Sharon
• Smith, Amy
• Hazrati, Lili-Naz
• Kresak, Jesse
• Gino, Somers
• Tan, Enrica
• Morales, Andres
• Santa-Maria, Vicente
• Hawkins, Cynthia
• Bartels, Ute
• Stephens, Derek
• Nobusawa, Sumihito
• Dufour, Christelle
• Bourdeaut, Franck
• Andre, Nicolas
• Bouffet, Eric
• Huang, Annie

Titel

ETMR-22. TITLE: DEFINING THE CLINICAL AND PROGNOSTIC LANDSCAPE OF EMBRYONAL TUMORS WITH MULTI-LAYERED ROSETTES (ETMRs), A RARE BRAIN TUMOR REGISTRY (RBTC) STUDY

Ist Teil von

• Neuro-oncology (Charlottesville, Va.), 2020-12, Vol.22 (Supplement_3), p.iii327-iii328

Ort / Verlag

US: Oxford University Press

Erscheinungsjahr

2020

Quelle

Oxford Journals 2020 Medicine

Beschreibungen/Notizen

• Abstract ETMR, an aggressive disease characterised by C19MC alterations, were previously categorised as various histologic diagnoses. The clinical spectrum and impact of conventional multi-modal therapy on this new WHO diagnostic category remains poorly understood as a majority of ~200 cases reported to date lack molecular confirmation. We undertook comprehensive clinico-pathologic studies of a large molecularly confirmed cohort to improve disease recognition and treatment approaches. Amongst 623 CNS-PNETs patients enrolled in the RBTC registry, 159 primary ETMRs were confirmed based on a combination of FISH (125), methylation analysis (88), SNP and RNAseq (32) analyses; 91% had C19MC amplification/gains/fusions, 9% lacked C19MC alterations but had global methylation features of ETMR NOS. ETMRs arose in young patients (median age 26 months) predominantly as localized disease (M0-72%, M2-3 -18%) at multiple locations including cerebrum (60%) cerebellum (18%), midline structures (6%); notably 10% were brainstem primaries mimicking DIPG. Uni-and multivariate analyses of clinical and treatment details of curative regimens available for 110 patients identified metastatic disease (p=0.002), brainstem locations(p=0.005), extent of surgery, receipt of multi-modal therapy including high dose chemotherapy and radiation (P<0.001) as significant treatment prognosticators, while C19MC status, age and gender were non-significant risk factors. Analyses of events in all patients showed respective EFS at 3 and 12 months of 84%(95%CI:77–91) and 37%(95%CI:20–41) and 4yr OS of 27%(95%CI:18–37) indicating despite intensified therapies ETMR is a rapidly progressive and fatal disease. Our comprehensive data on the largest cohort of molecularly-confirmed ETMRs provides a critical framework to guide current clinical management and development of clinical trials.