Chemotherapy

Recurrent CNS embryonal tumors can respond to chemotherapeutic agents used singularly or in combination, including cyclophosphamide, cisplatin, carboplatin, lomustine, etoposide, topotecan, temozolomide, the combination of irinotecan and temozolomide with or without bevacizumab, and antiangiogenic metronomic therapy.[9,17-27]; [28-30][Level of evidence B4] Approximately 30% to 50% of these patients have objective responses to conventional chemotherapy, but long-term disease control is rare.

For select patients with recurrent medulloblastoma—primarily infants and young children who were treated at the time of diagnosis with chemotherapy alone and who developed local recurrence—long-term disease control may be obtained after further treatment with chemotherapy plus local radiation therapy. This potential may be greatest in patients who are able to undergo complete resection of the recurrent disease.[31][Level of evidence B4]; [32][Level of evidence C1]

In a St. Jude Children’s Research Hospital study (SJYC07 [NCT00602667]), 29 patients with progressive disease received CSI (median dose, 36 Gy; interquartile range, 36–36). Of these 29 patients, 18 (62%) were alive at the time of publication, compared with 6 of 25 patients (24%) who did not receive CSI.[12][Level of evidence B4]