Researchers from France have reported that a high-dose regimen of busulfan and thiotepa supported by autologous stem cells followed by local radiotherapy resulted in a 69% 5-year survival in children with medulloblastoma that had progressed after initial surgery or surgery plus chemotherapy. The details of this study appeared in the July 1, 2007 issue of Cancer.

Medulloblastoma is a relatively rare brain tumor in children and has a grave prognosis. Treatment usually consists of maximum surgery followed by chemotherapy and radiation therapy. One of the major problems with this intensive therapy approach is brain damage (leukoencephalopathy), which is thought to be predominantly due to radiation. Because of the risk of leukoencephalopathy many children with medulloblastoma are often treated initially with chemotherapy. A recent multi-center trial has reported that children with average and high-risk medulloblastoma have improved survivals following post-operative risk-adapted radiotherapy and 4 cycles of high-dose cyclophosphamide and cisplatin with peripheral blood stem cell support (see related News).

The current study included 27 children with medulloblastoma who had disease progression after initial treatment with surgery and 12 had progressed after surgery and adjuvant chemotherapy. All were treated with high-dose busulfan and thiotepa with autologous stem cell support. The main toxicity of this regimen was veno-occlusive disease of the liver which occurred in one third of patients. Five-year survival of all 39 patients was 69%. For the 27 patients who were treated for a local recurrence, the 5-year survival was 77%. The 5 year survival for the 12 patients who progressed after chemotherapy was 50%.

Comments: These data suggest that high-dose busulfan and thiotepa and local radiation is an active regimen for medulloblastoma.

Reference:  Ridola V, Grill J, Doz F, et al. High-dose chemotherapy with autologous stem cell rescue followed by posterior fossa irradiation for local medulloblastoma recurrence or progression after conventional chemotherapy. Cancer. 2007; 110:156-163.

Related News:

Radiotherapy and Sequential Autologous Stem Cell Transplants May Improve Outcomes of Pediatric Medulloblastoma (10/4/2006)

Chemotherapy Alone is Promising for Pediatric Medulloblastoma (3/11/2005)