Journal de transplantation clinique et expérimentale

Abstrait

Haploidentical Stem Cell Transplantation and Stem Cell Mobilization for Relapsed Neuroblastoma

Michael Holzer

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.