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SHR Neuro Krebs Kardio Lipid Stoffw Microb

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; 24(5):1005-1012 Doi: 10.1016/j.bbmt.2017.12.805 [OPEN ACCESS]
Web of Science PubMed FullText FullText_MUG

Co-Autor*innen der Med Uni Graz: Urban Ernst-Christian
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Abstract:: 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 I/II 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 × 10⁶/kg T/B cell-depleted CD34⁺ stem cells with 68 × 10³/kg residual T cells and 107 × 10⁶/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 SCT relapsed within 1 year. Grade II 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. Copyright © 2018. Published by Elsevier Inc.
Find related publications in this database (Keywords): Neuroblastoma; Allogeneic stem cell transplantation; Haploidentical; Graft-versus-host disease; Transplantation-related mortality; Graft-versus-tumor effect