Advanced

Prognostic Value of PAX-FKHR Fusion Status in Alveolar Rhabdomyosarcoma: A Report From the Cooperative Soft Tissue Sarcoma Study Group (CWS)

Stegmaier, Sabine; Poremba, Christopher; Schaefer, Karl-Ludwig; Leuschner, Ivo; Kazanowska, Bernarda; Békássy, Albert LU ; Bielack, Stefan S.; Klingebiel, Thomas and Koscielniak, Ewa (2011) In Pediatric Blood & Cancer 57(3). p.406-414
Abstract
Background. Alveolar Rhabdomyosarcomas (RMA) are characterized by chromosomal translocations, fusing the PAX3 or PAX7 gene with FKHR in about 85%. Previous studies have suggested that the fusion type is associated with prognosis. In order to investigate the predictive value of the PAX-FKHR fusion status on disease outcome of patients with RMA treated in the CWS trials we performed a retrospective analysis. Procedure. Between 1986 and 2004, out of 446 patients with RMA treated in four consecutive CWS trials, tumor samples from 126 patients were available for RT-PCR analysis. Survival depending on fusion status in context with known clinical risk-factors was analyzed. Results. Out of 126 samples, 121 had adequate quality for PAX-FKHR fusion... (More)
Background. Alveolar Rhabdomyosarcomas (RMA) are characterized by chromosomal translocations, fusing the PAX3 or PAX7 gene with FKHR in about 85%. Previous studies have suggested that the fusion type is associated with prognosis. In order to investigate the predictive value of the PAX-FKHR fusion status on disease outcome of patients with RMA treated in the CWS trials we performed a retrospective analysis. Procedure. Between 1986 and 2004, out of 446 patients with RMA treated in four consecutive CWS trials, tumor samples from 126 patients were available for RT-PCR analysis. Survival depending on fusion status in context with known clinical risk-factors was analyzed. Results. Out of 126 samples, 121 had adequate quality for PAX-FKHR fusion status analysis. PAX-FKHR fusions were detected in 101 samples: 60% PAX3-FKHR and 24% PAX7-FKHR fusions, 17% were fusion-negative. There was no significant difference in survival between patients with PAX3-FKHR versus PAX7-FKHR positive tumors. The fusion transcript negative cohort showed a more favorable outcome than the fusion transcript positive cohort among patients with metastatic disease. From the established clinical risk-factors none was associated with a significantly higher risk of failure or death in a multivariate analysis. Conclusions. PAX-FKHR fusion type was not a significant predictor for survival in our analysis. Moreextensive molecular analyses are needed to identify features with prognostic relevance and useful therapeutic impact. Pediatr Blood Cancer 2011; 57: 406-414. (C) 2011 Wiley-Liss, Inc. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
PAX-FKHR, prognosis, rhabdomyosarcoma, RT-PCR, translocation
in
Pediatric Blood & Cancer
volume
57
issue
3
pages
406 - 414
publisher
John Wiley and Sons Inc.
external identifiers
  • wos:000293272800010
  • scopus:79958841531
ISSN
1545-5017
DOI
10.1002/pbc.22958
language
English
LU publication?
yes
id
09352edb-13f3-4abe-b55d-cf0ab6af95e3 (old id 2071832)
date added to LUP
2011-09-02 08:29:59
date last changed
2017-04-09 03:18:50
@article{09352edb-13f3-4abe-b55d-cf0ab6af95e3,
  abstract     = {Background. Alveolar Rhabdomyosarcomas (RMA) are characterized by chromosomal translocations, fusing the PAX3 or PAX7 gene with FKHR in about 85%. Previous studies have suggested that the fusion type is associated with prognosis. In order to investigate the predictive value of the PAX-FKHR fusion status on disease outcome of patients with RMA treated in the CWS trials we performed a retrospective analysis. Procedure. Between 1986 and 2004, out of 446 patients with RMA treated in four consecutive CWS trials, tumor samples from 126 patients were available for RT-PCR analysis. Survival depending on fusion status in context with known clinical risk-factors was analyzed. Results. Out of 126 samples, 121 had adequate quality for PAX-FKHR fusion status analysis. PAX-FKHR fusions were detected in 101 samples: 60% PAX3-FKHR and 24% PAX7-FKHR fusions, 17% were fusion-negative. There was no significant difference in survival between patients with PAX3-FKHR versus PAX7-FKHR positive tumors. The fusion transcript negative cohort showed a more favorable outcome than the fusion transcript positive cohort among patients with metastatic disease. From the established clinical risk-factors none was associated with a significantly higher risk of failure or death in a multivariate analysis. Conclusions. PAX-FKHR fusion type was not a significant predictor for survival in our analysis. Moreextensive molecular analyses are needed to identify features with prognostic relevance and useful therapeutic impact. Pediatr Blood Cancer 2011; 57: 406-414. (C) 2011 Wiley-Liss, Inc.},
  author       = {Stegmaier, Sabine and Poremba, Christopher and Schaefer, Karl-Ludwig and Leuschner, Ivo and Kazanowska, Bernarda and Békássy, Albert and Bielack, Stefan S. and Klingebiel, Thomas and Koscielniak, Ewa},
  issn         = {1545-5017},
  keyword      = {PAX-FKHR,prognosis,rhabdomyosarcoma,RT-PCR,translocation},
  language     = {eng},
  number       = {3},
  pages        = {406--414},
  publisher    = {John Wiley and Sons Inc.},
  series       = {Pediatric Blood & Cancer},
  title        = {Prognostic Value of PAX-FKHR Fusion Status in Alveolar Rhabdomyosarcoma: A Report From the Cooperative Soft Tissue Sarcoma Study Group (CWS)},
  url          = {http://dx.doi.org/10.1002/pbc.22958},
  volume       = {57},
  year         = {2011},
}