Journal
BRITISH JOURNAL OF CANCER
Volume 109, Issue 10, Pages 2523-2532Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/bjc.2013.630
Keywords
rhabdomyosarcoma; allogeneic haematopoietic stem cell transplantation; graft-vs-tumour effect; reduced intensity conditioning; myeloablative conditioning; donor lymphocyte infusion
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Funding
- Wilhelm Sander-Stiftung [2006.109.1]
- Else Kroner-Fresenius-Stiftung [P31/08//A123/07]
- BMBF (TranSarNet FK) [01GM0870, 01GM1104B]
- Deutsche Forschungsgemeinschaft (DFG) [GR3728/2-1]
- AmGen and Chugai and the Deutsche Kinderkrebsstiftung [DKS 2010.07]
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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.
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