Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz

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Gewählte Publikation:

SHR Neuro Krebs Kardio Lipid

Illhardt, T; Toporski, J; Feuchtinger, T; Turkiewicz, D; Teltschik, HM; Ebinger, M; Schwarze, CP; Holzer, U; Lode, HN; Albert, MH; Gruhn, B; Urban, C; Dykes, JH; Teuffel, O; Schumm, M; Handgretinger, R; Lang, P.
Haploidentical Stem Cell Transplantation for Refractory/Relapsed Neuroblastoma.
Biol Blood Marrow Transplant. 2018;
PubMed FullText FullText_MUG


Autor/innen der Med Uni Graz:
Urban Ernst-Christian

Dimensions Citations:

Plum Analytics:
Pediatric patients with refractory or relapsed metastatic neuroblastoma (NBL) have a poor prognosis despite autologous stem cell transplantation (SCT). Allogeneic SCT from haploidentical donors had remarkable alloreactive effects in leukemia patients; hence, we evaluated this approach in children with very high-risk NBL. We analyzed data from two prospective phase I/II trials. 26 patients with refractory (n=5) or metastatic relapsed (n=20) or locally relapsed MYCN+ (n=1) neuroblastoma received a median of 17x106/kg T/B cell depleted CD34+ stem cells with 68x103/kg residual T cells and 107x106/kg NK cells. The conditioning regimen comprised melphalan, fludarabine, thiotepa, OKT3 and short course mycophenolate mofetil posttransplant. Engraftment occurred in 96%. EFS and OS at 5 years were 19% and 23%, respectively. No transplant-related mortality was observed, and single cause of death was progression/subsequent relapse. Median follow-up was 8.1 years. Patients in complete remission before SCT had a significantly better prognosis than those with residual tumor load (p < 0.01). All patients with progressive disease before SCT relapsed within 1 year. Grades II / III acute GvHD were found in 31% and 12%, respectively. Chronic limited / extensive GvHD occurred in 28% and 10%. Haploidentical SCT is a feasible treatment option that can induce long-term remission in some patients with tolerable side effects and may enable further posttransplant therapeutic strategies based on the donor-derived immune system. Copyright © 2018. Published by Elsevier Inc.

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