Abstract

Despite autologous stem cell transplantation, paediatric patients with refractory or relapsed metastatic neuroblastoma (NBL) have a poor prognosis (SCT). We tested this strategy in kids with very high risk NBL since allogeneic SCT from a haploidentical donor has an impressive alloreactive effect in leukaemia patients. Data from two prospective phase I/II trials were examined. A median of 17 106/kg T/B cell-depleted CD34+ stem cells with 68 103/kg residual T cells and 107 106/kg natural killer cells were given to 26 patients with refractory (n = 5), metastatic relapsed (n = 20), or locally relapsed (n = 1) MYCN-positive NBL. Melphalan, fludarabine, thiotepa, OKT3, and a brief course of mycophenolate mofetil were all included in the conditioning regimen. 96 percent of the patients experienced engraftment. overall survival and event-free Survival rates at 5 years were 19 and 23%, respectively. The lone fatality was brought about by progression or a subsequent relapse; no transplantation-related mortality was noted.

Keywords: Melphalan; Fludarabine; Thiotepa; Mycophenolate