Intensive pediatric chemotherapy regimen (PNET HR+5) in adult high-risk medulloblastoma and pinealoblastoma patients

High-risk medulloblastoma (HRMB) is rare in adults. The 5-year overall survival rate is less than 60%. We present here a retrospective analysis of adults treated with an intensive pediatric chemo-radiotherapy regimen PNET HR+5: NCT00936156. 18 patients over the age of 20 (range, 20-33 years) with HRMB (n = 13), pinealoblastoma (n = 4) and central nervous system embryonal tumor (n = 1) were treated with 2 courses of carboplatin-etoposide followed by 2 courses of high-dose thiotepa (HDT) with autologous hematopoietic stem-cell rescue. A craniospinal irradiation (CSI) (36 Gy craniospinal axis then a boost of 18 Gy to the primary tumor site) was then initiated within 150 days of surgery, completed with 6 cycles of temozolomide; the axis irradiation was not mandatory for non-metastatic pinealoblastoma. We observed no progression under chemotherapy and no toxic death. Four patients received only 1 HDT. Two non-metastatic pinealoblastoma received only focal irradiation. One medulloblastoma received only 25 Gy on axis. 56% (10/18) received 6 cycles of temozolomide. No long-term toxicity was recorded. Median time between surgery and CSI was 175 days (range, 115-250). With a median follow-up of 6.0 years (range, 2.6-9), the progression-free survival and overall survival rates for medulloblastoma were respectively 65% (95% CI: 31-86%) and 76% (95% CI: 42-91%) at 5 years. The PNET HR+5 regimen showed promising results in an adult population, with a meaningful improvement in progression-free survival and overall survival in patients with HRMB.