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

Research output: Contribution to journalArticle

Abstract

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
Volume109
Issue number10
DOIs
Publication statusPublished - Nov 12 2013

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

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    Thiel, U., Koscielniak, E., Blaeschke, F., Grunewald, T. G. P., Badoglio, M., Diaz, M. A., Paillard, C., Prete, A., Ussowicz, M., Lang, P., Fagioli, F., Lutz, P., Ehninger, G., Schneider, P., Santucci, A., Bader, P., Gruhn, B., Faraci, M., Antunovic, P., ... Burdach, S. (2013). Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma: A retrospective assessment. British Journal of Cancer, 109(10), 2523-2532. https://doi.org/10.1038/bjc.2013.630