Overall survival (OS) was 70.5%. Disease free survival (DFS) was 54.6%. There was no statistically significant difference between treatment groups in either OS or DFS. Overall survival in patients younger than one year was higher (88.2%). There were significantly more patients with 100% donor chimerism transplanted with flu/treo/tt compared with flu/treo or treo/cy (94.7% compared to 66.7% and 16.7%, respectively). Further prospective studies are required to determine the optimal treosulfan-based preparative regimen for children with non-malignant diseases. Pediatr Blood Cancer 2015;62:299-304. © 2014 Wiley Periodicals, Inc.

Contemporary protocols for the treatment of malignant brain tumors such as medulloblastoma (MB) in children, often involve craniospinal irradiation (CSI) at diagnosis followed by serial courses of high dose chemotherapy and autologous hematopoietic stem cell support. Patients often require several pheresis procedures in order to collect sufficient stem cells for this type of treatment, particularly if they have already had CSI. We describe the successful mobilization, collection and subsequent transplant of a 7-year-old female with medulloblastoma after recent CSI using granulocyte colony stimulating factor (G-CSF) and the CXCR4 antagonist AMD3100 after a failed previous mobilization attempt using G-CSF alone.

Discontinuation of CNIs results in neurologic improvement in most cases, but superior alternative immune modulatory treatment is needed to prevent progression of established GVHD.

Percutaneous placement of radial artery catheters can be rapidly and safely performed in very small infants and in children with difficult venous access. This technique provides a reliable platform for efficient PBSCC.