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)

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, M. C. Frühwald

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Abstract

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.

Original languageEnglish
Pages (from-to)370-375
Number of pages6
JournalBone Marrow Transplantation
Volume49
Issue number3
DOIs
Publication statusPublished - márc. 1 2014

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ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Benesch, M., Bartelheim, K., Fleischhack, G., Gruhn, B., Schlegel, P. G., Witt, O., Stachel, K. D., Hauch, H., Urban, C., Quehenberger, F., Massimino, M., Pietsch, T., Hasselblatt, M., Giangaspero, F., Kordes, U., Schneppenheim, R., Hauser, P., Klingebiel, T., & Frühwald, M. C. (2014). 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 Transplantation, 49(3), 370-375. https://doi.org/10.1038/bmt.2013.208