Prognostic factors and treatment results for supratentorial primitive neuroectodermal tumors in children using radiation and chemotherapy: A Childrens Cancer Group randomized trial

Purpose: To determine clinical characteristics and response to treatment for children with supratentorial primitive neuroectodermal tumors (S-PNETs). Patients and Methods: After surgery and staging, 55 patients aged 1.5 to 19.3 years with S-PNETs were randomized to receive craniospinal radiotherapy (RT) followed by eight cycles of 1-(2-chloro-ethyl)-3-cyclohexyl-nitrosourea (CCNU), vincristine (VCR), and prednisone (standard treatment) or two cycles of 8-in-1 chemotherapy followed by RT and then eight additional cycles of 8- in-1. Results: Three-year Kaplan-Meier estimates (estimate ± SE) of survival and progression-free survival (PFS) rates for patients with confirmed diagnoses of S-PNET were 57% ± 8% and 45% ± 8%, respectively; survival and PFS rates for children with PNETs located in the pineal region were 73% ± 12% and 61% ± 13%, respectively, and were significantly different from the other S-PNETs (P < .03). The 8-in-1 arm had greater toxicity than the standard-treatment arm. Distributions of PFS between the two treatment groups were not significantly different (P > .5). Other univariate prognostic factors that influenced PFS included metastasis (M) stage (P < .03: M₀ 50% ± 9% v M_1-4 0%) and age (P < .02: 1.5 to 2 years 25% ± 13% v ≥ 3 years 53% ± 9%). Conclusion: In this first randomized treatment trial for S-PNETs in children, no significant differences were detected between the two treatment groups. M₀ and pineal site of involvement were independent predictors of a better outcome. However, survival was better than previously reported.