HIGH-DOSE CHEMOTHERAPY WITH AUTOLOGOUS STEM-CELL RESCUE IN PATIENTS WITH RECURRENT AND HIGH-RISK PEDIATRIC BRAIN-TUMORS

Purpose: We treated 49 patients with recurrent or poor-prognosis CNS m alignancies with high-dose chemotherapy regimens followed by autologou s marrow rescue with or without peripheral-blood stem-cell augmentatio n to determine the toxicity of and event-free survival after these reg imens. patients and Methods: Nineteen patients had medulloblastomas, 1 2 had glial tumors, seven had pineoblastomas, five had ependymomas, th ree had primitive neuroectodermal tumors, two had germ cell tumors, an d one had fibrosarcoma. Thirty-seven received chemotherapy with cyclop hosphamide 1.5 g/m(2) daily x 4 and melphalan 25 to 60 mg/m(2) daily x 3. Nine received busulfan 37.5 mg/m(2) every 6 hours x 16 and melphal an 180 mg/m(2) (n = 7) or 140 mg/m(2) (n = 2). Three received carbopla tin 700 mg/m(2)/d on days -7, -5, and -3 and etoposide 500 mg/m(2)/d o n days -6, -4, and -2. All patients received standard supportive care. Results: Eighteen of 49 patients survive event-free 22+ to 55+ months [median, 33+) after transplantation, including nine of 16 treated bef ore recurrence and nine of 33 treated after recurrence. There was one transplant-related death from pulmonary aspergillosis. Of five patient s assessable for disease response, one had a partial remission (2 mont hs), one has had stable disease (55+ months), and three showed progres sion 2, 5, and 8 months after transplantation. Conclusion: The toxicit y of these regimens was tolerable. Certain patients with high-risk CNS malignancies may benefit from such a treatment approach. Subsequent t rials should attempt to determine which patients are most likely to be nefit from high-dose chemotherapy with autologous stem-cell rescue. (C ) 1997 by American Society of Clinical Oncology.