Prognostic Value of PAX-FKHR Fusion Status in Alveolar Rhabdomyosarcoma: A Report From the Cooperative Soft Tissue Sarcoma Study Group (CWS)
(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:
https://lup.lub.lu.se/record/2071832
- author
- Stegmaier, Sabine ; Poremba, Christopher ; Schaefer, Karl-Ludwig ; Leuschner, Ivo ; Kazanowska, Bernarda ; Békássy, Albert LU ; Bielack, Stefan S. ; Klingebiel, Thomas and Koscielniak, Ewa
- organization
- publishing date
- 2011
- 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 & Sons Inc.
- external identifiers
-
- wos:000293272800010
- scopus:79958841531
- pmid:21254373
- 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
- 2016-04-01 10:49:01
- date last changed
- 2022-04-20 06:30:45
@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}},
keywords = {{PAX-FKHR; prognosis; rhabdomyosarcoma; RT-PCR; translocation}},
language = {{eng}},
number = {{3}},
pages = {{406--414}},
publisher = {{John Wiley & 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}},
doi = {{10.1002/pbc.22958}},
volume = {{57}},
year = {{2011}},
}