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High-dose chemotherapy (HDCT) with auto-SCT in children with atypical teratoid/rhabdoid tumors (AT/RT): a report from the European Rhabdoid Registry (EU-RHAB)

A retrospective analysis of data from the European Rhabdoid Registry (EU-RHAB) was performed to describe the outcome of children with atypical teratoid/rhabdoid tumors (AT/RT) who underwent high-dose chemotherapy (HDCT) with auto-SCT. Nineteen patients (male, n=15; median age at diagnosis 21 months)...

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Main Authors: Benesch, Martin (Author) , Bartelheim, Kerstin (Author) , Fleischhack, G. (Author) , Gruhn, Bernd (Author) , Schlegel, Paul-Gerhardt (Author) , Witt, Olaf (Author) , Stachel, Klaus Daniel (Author) , Hauch, Holger (Author) , Urban, Christian (Author) , Quehenberger, F. (Author) , Massimino, M. (Author) , Pietsch, T. (Author) , Hasselblatt, Martin (Author) , Giangaspero, F. (Author) , Kordes, Uwe (Author) , Schneppenheim, Reinhard (Author) , Hauser, Peter (Author) , Klingebiel, Thomas (Author) , Frühwald, Michael (Author)
Format: Article (Journal)
Language:English
Published: 13 January 2014
In: Bone marrow transplantation
Year: 2014, Volume: 49, Issue: 3, Pages: 370-375
ISSN:1476-5365
DOI:10.1038/bmt.2013.208
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/bmt.2013.208
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/bmt2013208
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Author Notes:M. Benesch, K. Bartelheim, G. Fleischhack, B. Gruhn, P.G. Schlegel, O. Witt, K.D. Stachel, H. Hauch, C. Urban, F. Quehenberger, M. Massimino, T. Pietsch, M. Hasselblatt, F. Giangaspero, U. Kordes, R. Schneppenheim, P. Hauser, T. Klingebiel and M.C. Frühwald
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Summary:A retrospective analysis of data from the European Rhabdoid Registry (EU-RHAB) was performed to describe the outcome of children with atypical teratoid/rhabdoid tumors (AT/RT) who underwent high-dose chemotherapy (HDCT) with auto-SCT. Nineteen patients (male, n=15; median age at diagnosis 21 months) were identified. Nine patients presented with metastatic disease at diagnosis. A partial or subtotal resection was achieved in 11, a total resection in five and a biopsy in three patients. Patients received a median of six chemotherapy cycles prior to HDCT. Additional radiotherapy was performed in 14 patients (first-line, n=9; following progression, n=5). Six patients underwent tandem auto-SCT. Disease status before HDCT was CR in six, PR in eight, stable disease in two and progressive disease (PD) in two patients (data missing, n=1). With a median follow-up of 16 months, 14 patients progressed. Estimated progression-free and OS at 2 years were 29% (±11%) and 50% (±12%), respectively. At last follow-up, eight patients were alive (first CR, n=4; second CR, n=2; PR, n=1; PD, n=1). Eleven patients died of PD. Median time-to-progression was 14 months. Selected patients with AT/RT might benefit from HDCT with radiotherapy. The definitive impact of this treatment modality has to be evaluated prospectively in a randomized trial.
Item Description:Gesehen am 16.09.2020
Physical Description:Online Resource
ISSN:1476-5365
DOI:10.1038/bmt.2013.208

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