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

作者：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; Styczynski J; Krueger W H; Castagna L; Rohrlich P; Ouachee Chardin M; Salmon A; Peters C; Bregni M; Burdach S

来源：British Journal of Cancer, 2013, 109(10): 2523-2532.

DOI：10.1038/bjc.2013.630

• rhabdomyosarcoma
• allogeneic haematopoietic stem cell transplantation
• graft-vs-tumour effect
• reduced intensity conditioning
• myeloablative conditioning
• donor lymphocyte infusion

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. %26lt;br%26gt;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. %26lt;br%26gt;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. %26lt;br%26gt;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.

• 出版日期2013-11-12
• 单位Perugia