High Dose Chemotherapy with Autologous Peripheral Blood Stem Cell Transplantation in Patients with Medulloblastoma/Primitive Neuroectodermal Tumor / 대한소아혈액종양학회지

ABSTRACT

PURPOSE: To improve survival and/or to avoid radiotherapy, high dose chemotherapy (HDCT) with autologous peripheral blood stem cell transplantation (PBSCT) was given to patients with recurrent or high risk medulloblastoma (MB)/primitive neuroectodermal tumor (PNET) as well as patients younger than 3 years of age. METHODS: Six patients (3 recurrent, 1 high risk, 2 younger than 3 years; 5 MBs and 1 PNET) received single or double HDCT and PBSCT with or without immunotherapy using interleukin-2. Chemotherapeutic regimen in the first HDCT included cyclophosphamide (1,500 mg/m2/ day for 4 days) and melphalan (60 mg/m2/day for 3 days). Chemotherapeutic regimen in the second HDCT included carboplatin (400 mg/m2/day for 3 days), thiotepa (250 mg/ m2/day for 3 days), and etoposide (200 mg/m2/day for 3 days). RESULTS: Nine HDCTs were applied in 6 patients. Three double HDCTs were rescued with peripheral blood stem cells collected during single round leukapheresis. Rapid hematologic recovery occurred in 4 patients. Engraft failure occurred in 1 patient and delayed granulocyte recovery and platelet engraft failure occurred in 1 patient. Three patients who had minimal disease before HDCT had event free survival for 7~18 months after HDCT. Tumor relapsed 8 and 12 months after single HDCT in 2 patients among 3 patients with recurrent MB/PNET. One patient with recurrent MB died due to engraft failure and sepsis. CONCLUSION: HDCT with autologous PBSCT is expected to improve survival of patients with poor prognosis MB/PNET including younger patients less than 3 years. Subsequent trials with larger number of patients and long-term follow-up are needed.