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Illhardt, Toni; Toporski, Jacek; Feuchtinger, Tobias; Turkiewicz, Dominik; Teltschik, Heiko-Manuel; Ebinger, Martin; Schwarze, Carl -Philipp; Holzer, Ursula; Lode, Holger N.; Albert, Michael H.; Gruhn, Bernd; Urban, Christian; Dykes, Josefina H.; Teuffel, Oliver; Schumm, Michael; Handgretinger, Rupert and Lang, Peter (2018): Haploidentical Stem Cell Transplantation for Refractory/Relapsed Neuroblastoma. In: Biology of Blood and Marrow Transplantation, Vol. 24, No. 5: pp. 1005-1012

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Pediatric patients with refractory or relapsed metastatic neuroblastoma (NBL) have a poor prognosis despite autologous stem cell transplantation (SCT). Allogeneic SCT from a haploidentical donor has a remarkable alloreactive effect in patients with leukemia;thus, we evaluated this approach in children with very high risk NBL. We analyzed data from 2 prospective phase 1/11 trials. A total of 26 patients with refractory (n = 5), metastatic relapsed (n = 20), or locally relapsed MYCN-positive (n =1) NBL received a median of 17 x 106/kg T/13 cell-depleted CD34. stem cells with 68 x 10(3)/kg residual T cells and 10(7) x 10(6)/kg natural killer cells. The conditioning regimen comprised melphalan, fludarabine, thiotepa, OKT3, and a short course of mycophenolate mofetil post-transplantation. Engraftment occurred in 96% of the patients. Event-free survival and overall survival at 5 years were 19% and 23%, respectively. No transplantation-related mortality was observed, and the single death was due to progression/subsequent relapse. The median duration of follow-up was 8.1 years. Patients in complete remission before SCT had a significantly better prognosis than those with residual tumor load (P <.01). All patients with progressive disease before so. relapsed within 1 year. Grade 11 and grade III acute graft-versus-host disease (GVHD) occurred in 31% and 12% of the patients, respectively. Chronic limited and extensive GVHD occurred in 28% and 10%, respectively. Our data indicate that haploidentical SCT is a feasible treatment option that can induce long-term remission in some patients with NBL with tolerable side effects, and may enable the development of further post-transplantation therapeutic strategies based on the donor-derived immune system.

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