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Pediatric Transplants

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 volume 49, pages 370–375 (2014)Cite this article

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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.

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Acknowledgements

We thank Petra Neumayer (EU-RHAB trial center Augsburg, Germany) for excellent data management and administrative support. EU-RHAB is supported by the Deutsche Kinderkrebsstiftung (German Childhood Cancer Foundation).

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Authors and Affiliations

  1. Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria

    M Benesch & C Urban

  2. Swabian Children’s Cancer Center, Children’s Hospital Augsburg, Augsburg, Germany

    K Bartelheim & M C Frühwald

  3. Pediatrics III, University Hospital of Essen, Essen, Germany

    G Fleischhack

  4. Department of Pediatrics, Jena University Hospital, Jena, Germany

    B Gruhn

  5. Department of Pediatric Hematology, Oncology and Neurooncology, University Children’s Hospital Würzburg, Würzburg, Germany

    P G Schlegel

  6. Department of Pediatric Oncology, Hematology, Immunology and Pneumonology, Children’s Hospital, University of Heidelberg, Heidelberg, Germany

    O Witt

  7. Children’s University Hospital, Friedrich-Alexander University Erlangen-Nürnberg, Erlangen, Germany

    K D Stachel

  8. Department of Pediatric Hematology and Oncology, Justus Liebig University Gießen, Gießen, Germany

    H Hauch

  9. Institute for Medical Statistics, Medical University of Graz, Graz, Austria

    F Quehenberger

  10. Department of Pediatrics, Fondazione IRCCS, Istituto Nazionale Tumori, Milan, Italy

    M Massimino

  11. Institute of Neuropathology, University of Bonn, Bonn, Germany

    T Pietsch

  12. Institute of Neuropathology, University Hospital Münster, Münster, Germany

    M Hasselblatt

  13. Department of Radiological, Oncological and Anatomo-pathological Sciences, University La Sapienza, Rome, Italy

    F Giangaspero

  14. IRCCS Neuromed, Pozzilli, Italy

    F Giangaspero

  15. Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

    U Kordes & R Schneppenheim

  16. Department of Pediatrics, Semmelweis University, Budapest, Hungary

    P Hauser

  17. Division of Pediatric Hematology and Oncology, Department of Pediatrics, J. W. Goethe University Children’s Hospital of Frankfurt, Frankfurt, Germany

    T Klingebiel

  18. Department of Pediatric Hematology and Oncology, University Children’s Hospital Münster, Münster, Germany

    M C Frühwald

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  1. M Benesch
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  2. K Bartelheim
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  19. M C Frühwald
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Correspondence to M Benesch or M C Frühwald.

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Benesch, M., Bartelheim, K., Fleischhack, G. et al. 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 49, 370–375 (2014). https://doi.org/10.1038/bmt.2013.208

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