title: Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma: a retrospective assessment.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Wilhelm Sander Sarcoma Unit, Department of Pediatrics, Roman Herzog Comprehensive Cancer Center, Klinikum rechts der Isar, Technische Universität München, 81664 München, Germany.
authors.forename: U
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authors.lastname: Thiel
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authors.lastname: Koscielniak
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authors.initials: F
authors.lastname: Blaeschke
authors.forename: T G P
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authors.lastname: Grunewald
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authors.lastname: Badoglio
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authors.lastname: Paillard
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authors.lastname: Prete
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authors.lastname: Ussowicz
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authors.lastname: Lang
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authors.lastname: Fagioli
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authors.lastname: Lutz
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authors.lastname: Ehninger
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authors.lastname: Schneider
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authors.lastname: Santucci
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authors.lastname: Gruhn
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authors.lastname: Faraci
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authors.lastname: Antunovic
authors.forename: J
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authors.lastname: Styczynski
authors.forename: W H
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authors.lastname: Krüger
authors.forename: L
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authors.lastname: Castagna
authors.forename: P
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authors.lastname: Rohrlich
authors.forename: M
authors.initials: M
authors.lastname: Ouachée-Chardin
authors.forename: A
authors.initials: A
authors.lastname: Salmon
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: M
authors.initials: M
authors.lastname: Bregni
authors.forename: S
authors.initials: S
authors.lastname: Burdach
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000453:epidemiology / Q000401:mortality; D012189:Retrospective Studies; D012208:Rhabdomyosarcoma / Q000401:mortality / Q000601:surgery*; D014184:Transplantation, Homologous; D055815:Young Adult