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. Styczynski; W. H. Krüger; L. Castagna; P. Rohrlich; M. Ouachée-Chardin; A. Salmon; C. Peters; M. Bregni; S. Burdach

doi:10.1038/bjc.2013.630

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

Medicine and Health

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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 language	English
Pages (from-to)	2523-2532
Number of pages	10
Journal	British Journal of Cancer
Volume	109
Issue number	10
DOIs	https://doi.org/10.1038/bjc.2013.630
State	Published - 12 Nov 2013

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10.1038/bjc.2013.630

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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

@article{8b6c99b9382240458f7f59c13b21fc0e,

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.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.",

author = "U. Thiel and E. Koscielniak and F. Blaeschke and Grunewald, {T. G.P.} and M. Badoglio and Diaz, {M. A.} and C. Paillard and A. Prete and M. Ussowicz and P. Lang and F. Fagioli and P. Lutz and G. Ehninger and P. Schneider and A. Santucci and P. Bader and B. Gruhn and M. Faraci and P. Antunovic and J. Styczynski and Kr{\"u}ger, {W. H.} and L. Castagna and P. Rohrlich and M. Ouach{\'e}e-Chardin and A. Salmon and C. Peters and M. Bregni and S. Burdach",

note = "Funding Information: This work was supported by by grants to SB from the Wilhelm Sander-Stiftung (2006.109.1), Else Kr{\"o}ner–Fresenius–Stiftung (SB and GR; P31/08//A123/07), BMBF (SB and GR; TranSarNet FK:01GM0870), the Deutsche Forschungsgemeinschaft (DFG, GR3728/2-1 to TG), AmGen and Chugai and the Deutsche Kinderkrebsstiftung (SB and GR; DKS 2010.07) and to SB, GR, and UT from the BMBF (TranSarNet 01GM1104B). We wish to thank all patients and their families as well as all data managers, physicians, and nurses for their contribution to this study. Petra Wolf is especially acknowledged for helpful advice in the statistical evaluation.",

year = "2013",

month = nov,

day = "12",

doi = "10.1038/bjc.2013.630",

language = "English",

volume = "109",

pages = "2523--2532",

journal = "British Journal of Cancer",

issn = "0007-0920",

publisher = "Nature Publishing Group",

number = "10",

}

Thiel, U, Koscielniak, E, Blaeschke, F, Grunewald, TGP, Badoglio, M, Diaz, MA, 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, Krüger, WH, Castagna, L, Rohrlich, P, Ouachée-Chardin, M, Salmon, A, Peters, C, Bregni, M & Burdach, S 2013, 'Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma: A retrospective assessment', British Journal of Cancer, vol. 109, no. 10, pp. 2523-2532. https://doi.org/10.1038/bjc.2013.630

TY - JOUR

T1 - Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma

T2 - A retrospective assessment

AU - Thiel, U.

AU - Koscielniak, E.

AU - Blaeschke, F.

AU - Grunewald, T. G.P.

AU - Badoglio, M.

AU - Diaz, M. A.

AU - Paillard, C.

AU - Prete, A.

AU - Ussowicz, M.

AU - Lang, P.

AU - Fagioli, F.

AU - Lutz, P.

AU - Ehninger, G.

AU - Schneider, P.

AU - Santucci, A.

AU - Bader, P.

AU - Gruhn, B.

AU - Faraci, M.

AU - Antunovic, P.

AU - Styczynski, J.

AU - Krüger, W. H.

AU - Castagna, L.

AU - Rohrlich, P.

AU - Ouachée-Chardin, M.

AU - Salmon, A.

AU - Peters, C.

AU - Bregni, M.

AU - Burdach, S.

N1 - Funding Information: This work was supported by by grants to SB from the Wilhelm Sander-Stiftung (2006.109.1), Else Kröner–Fresenius–Stiftung (SB and GR; P31/08//A123/07), BMBF (SB and GR; TranSarNet FK:01GM0870), the Deutsche Forschungsgemeinschaft (DFG, GR3728/2-1 to TG), AmGen and Chugai and the Deutsche Kinderkrebsstiftung (SB and GR; DKS 2010.07) and to SB, GR, and UT from the BMBF (TranSarNet 01GM1104B). We wish to thank all patients and their families as well as all data managers, physicians, and nurses for their contribution to this study. Petra Wolf is especially acknowledged for helpful advice in the statistical evaluation.

PY - 2013/11/12

Y1 - 2013/11/12

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=84887838962&partnerID=8YFLogxK

U2 - 10.1038/bjc.2013.630

DO - 10.1038/bjc.2013.630

M3 - Article

C2 - 24149176

AN - SCOPUS:84887838962

SN - 0007-0920

VL - 109

SP - 2523

EP - 2532

JO - British Journal of Cancer

JF - British Journal of Cancer

IS - 10

ER -

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

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