Pediatric intracranial ependymoma: correlating signs and symptoms at recurrence with outcome in the second prospective AIEOP protocol follow-up

Maura Massimino, Francesco Barretta, Piergiorgio Modena, Felice Giangaspero, Luisa Chiapparini, Alessandra Erbetta, Luna Boschetti, Manila Antonelli, Paolo Ferroli, Daniele Bertin, Emilia Pecori, Veronica Biassoni, Maria Luisa Garrè, Elisabetta Schiavello, Iacopo Sardi, Elisabetta Viscardi, Giovanni Scarzello, Maurizio Mascarin, Lucia Quaglietta, Giuseppe Cinalli, Lorenzo Genitori, Paola Peretta, Anna Mussano, Salvina Barra, Angela Mastronuzzi, Carlo Giussani, Carlo Efisio Marras, Rita Balter, Patrizia Bertolini, Assunta Tornesello, Milena La Spina, Francesca Romana Buttarelli, Antonio Ruggiero, Massimo Caldarelli, Geraldina Poggi, Lorenza Gandola

The aims of patients' radiological surveillance are to: ascertain relapse; apply second-line therapy; accrue patients in phase 1/2 protocols if second-line therapy is not standardized/curative; and assess/treat iatrogenic effects. To lessen the emotional and socioeconomic burdens for patients and families, we ideally need to establish whether scheduled radiological surveillance gives patients a better outcome than waiting for symptoms and signs to appear. We analyzed a prospective series of 160 newly-diagnosed and treated pediatric/adolescent patients with intracranial ependymoma, comparing patients with recurrent disease identified on scheduled MRI (the RECPT group; 34 cases) with those showing signs/symptoms of recurrent disease (the SYMPPT group; 16 cases). The median follow-up was 67 months. No significant differences emerged between the two groups in terms of gender, age, tumor grade/site, shunting, residual disease, or type of relapse (local, distant, or concomitant). The time to relapse (median 19 months; range 5-104) and the MRI follow-up intervals did not differ between the SYMPPT and RECPT groups. The presence of signs/symptoms was an unfavorable factor for overall survival (OS) after recurrence (5-year OS: 8% vs. 37%, p = 0.001). On multivariable analysis, an adjusted model confirmed a significantly worse OS in the SYMPPT than in the RECPT patients. Symptomatic relapses carried a significantly worse survival for ependymoma patients than recurrences detected by MRI alone. It would therefore be desirable to identify recurrences before symptoms develop. Radiological follow-up should be retained in ependymoma patient surveillance because there is a chance of salvage treatment for relapses found on MRI.