Graft versus tumor effect in the brain of a child with recurrent metastatic medulloblastoma

Marrow ablative chemotherapy (MAC) with autologous hematopoietic stem cell transplantation (HSCT) is limited by poor bone marrow reserve after chemotherapy and/or radiotherapy, and the extent of bone/bone marrow disease. We report a child with recurrent metastatic medulloblastoma who received an allogeneic HSCT while in relapse and subsequently achieved radiological resolution of disease and favorable marrow minimal residual disease (MRD) response. Disease recurred intra-cranially at 304 days post-HSCT. Tumor biopsy 488 days post-HSCT showed infiltration with donor lymphocytes demonstrating graft-versus-tumor (GVT) effect. The patient remained alive >2 years post-HSCT. Allogeneic HSCT may be a consideration for high-risk recurrent medulloblastoma.