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Benesch, M; Bartelheim, K; Fleischhack, G; Gruhn, B; Schlegel, PG; Witt, O; Stachel, KD; Hauch, H; Urban, C; Quehenberger, F; Massimino, M; Pietsch, T; Hasselblatt, M; Giangaspero, F; Kordes, U; Schneppenheim, R; Hauser, P; Klingebiel, T; Frühwald, MC.
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).
Bone Marrow Transplant. 2014; 49(3):370-375
Doi: 10.1038/bmt.2013.208
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- Leading authors Med Uni Graz
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Benesch Martin
- Co-authors Med Uni Graz
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Quehenberger Franz
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Urban Ernst-Christian
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- Abstract:
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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.
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atypical teratoid/rhabdoid tumors
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auto-SCT
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children