Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma: A retrospective assessment

U. Thiel, E. Koscielniak, F. Blaeschke, T. G.P. Grunewald, M. Badoglio, M. A. Diaz, C. Paillard, A. Prete, M. Ussowicz, P. Lang, F. Fagioli, P. Lutz, G. Ehninger, P. Schneider, A. Santucci, P. Bader, B. Gruhn, M. Faraci, P. Antunovic, J. StyczynskiW. H. Krüger, L. Castagna, P. Rohrlich, M. Ouachée-Chardin, A. Salmon, C. Peters, M. Bregni, S. Burdach

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22 Scopus citations


Background:Allogeneic haematopoietic stem cell transplantation (allo-SCT) may provide donor cytotoxic T cell-/NK cell-mediated disease control in patients with rhabdomyosarcoma (RMS). However, little is known about the prevalence of graft-vs-RMS effects and only a few case experiences have been reported.Methods:We evaluated allo-SCT outcomes of 30 European Group for Blood and Marrow Transplantation (EBMT)-registered patients with advanced RMS regarding toxicity, progression-free survival (PFS) and overall survival (OS) after allo-SCT. Twenty patients were conditioned with reduced intensity and ten with high-dose chemotherapy. Twenty-three patients were transplanted with HLA-matched and seven with HLA-mismatched grafts. Three patients additionally received donor lymphocyte infusions (DLIs). Median follow-up was 9 months.Results:Three-year OS was 20% (s.e.±8%) with a median survival time of 12 months. Cumulative risk of progression was 67% (s.e.±10%) and 11% (s.e.±6%) for death of complications. Thirteen patients developed acute graft-vs-host disease (GvHD) and five developed chronic GvHD. Eighteen patients died of disease and four of complications. Eight patients survived in complete remission (CR) (median: 44 months). No patients with residual disease before allo-SCT were converted to CR.Conclusion:The use of allo-SCT in patients with advanced RMS is currently experimental. In a subset of patients, it may constitute a valuable approach for consolidating CR, but this needs to be validated in prospective trials.

Original languageEnglish
Pages (from-to)2523-2532
Number of pages10
JournalBritish Journal of Cancer
Issue number10
StatePublished - 12 Nov 2013


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