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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Hauptverfasser: Benesch, Martin (VerfasserIn) , Bartelheim, Kerstin (VerfasserIn) , Fleischhack, G. (VerfasserIn) , Gruhn, Bernd (VerfasserIn) , Schlegel, Paul-Gerhardt (VerfasserIn) , Witt, Olaf (VerfasserIn) , Stachel, Klaus Daniel (VerfasserIn) , Hauch, Holger (VerfasserIn) , Urban, Christian (VerfasserIn) , Quehenberger, F. (VerfasserIn) , Massimino, M. (VerfasserIn) , Pietsch, T. (VerfasserIn) , Hasselblatt, Martin (VerfasserIn) , Giangaspero, F. (VerfasserIn) , Kordes, Uwe (VerfasserIn) , Schneppenheim, Reinhard (VerfasserIn) , Hauser, Peter (VerfasserIn) , Klingebiel, Thomas (VerfasserIn) , Frühwald, Michael (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 13 January 2014
In: Bone marrow transplantation
Year: 2014, Jahrgang: 49, Heft: 3, Pages: 370-375
ISSN:1476-5365
DOI:10.1038/bmt.2013.208
Online-Zugang:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/bmt.2013.208
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/bmt2013208
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Verfasserangaben: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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Zusammenfassung: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.
Beschreibung:Gesehen am 16.09.2020
Beschreibung:Online Resource
ISSN:1476-5365
DOI:10.1038/bmt.2013.208