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Tumor control and QoL outcomes of very young children with atypical teratoid/rhabdoid Tumor treated with focal only chemo-radiation therapy using pencil beam scanning proton therapy

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Abstract

The aim of this analysis was to assess the early clinical results of pencil beam scanning proton therapy (PT) in the treatment of young children with non-metastatic atypical teratoid/rhabdoid tumor (ATRT) of the CNS. Fifteen children (male, n = 8, 53 %) were treated with PT between May 2008 and January 2013. Mean age at diagnosis was 17.4 ± 7.0 months. The localization was infratentorial in 9 (60 %) patients. Gross total resection of the primary tumors was achieved in 7 (47 %) patients. The dose administered focally under sedation was 54 Gy (RBE). After a median follow-up of 33.4 months (range 9.7–69.2), 3 (20 %), 4 (27 %) and 2 (13 %) patients presented with local failure (LF), distant brain failure (DBF) and spinal failure (SF), respectively. Six patients died, all of tumor progression. The 2-year overall- and progression-free survival was 64.6 and 66.0 %. Tumor location (supratentorial) and the extent of surgical resection (non-gross total resection) were negative prognostic factors for both OS and PFS. PT was well tolerated. No grade >2 acute toxicity was observed. The estimated 2-year toxicity-free survival was 90 %. As assessed by the PedsQoL proxy, no decrease in QoL was observed after PT. We conclude that PBS PT is an effective treatment for young children with ATRT. After PT, with or without concomitant chemotherapy, two third of the patients survived >2 years. Acute toxicity was manageable. Longer follow-up and larger numbers of patients are needed to assess long-term outcomes and treatment-induced toxicity.

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Acknowledgments

We thank Carmen Teske (University of Münster), Anna Wiener Wellauer (PSI), April Siegwolf (PSI) and Beate Schulz (PSI) for providing data management.

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The author & co-authors have no potential conflict of interest.

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Authors and Affiliations

  1. Center for Proton Therapy, Paul Scherrer Institute, WPTA 144, CH-5232, Villigen West, Switzerland

    Damien C. Weber, Robert Malyapa, Francesca Albertini, Ulrike Kliebsch, Lorentzos Mikroutsikos, Petra Morach, Alessandra Bolsi, Tony Lomax & Ralf Schneider

  2. University of Zürich, Zurich, Switzerland

    Damien C. Weber

  3. Department of Radiation Oncology, Geneva University Hospital, Geneva, Switzerland

    Carmen Ares

  4. Department of Pediatric Hematology and Oncology, University, Hospital Münster, Münster, Germany

    Gabriele Calaminus

  5. Department of Physics, Swiss Institute of Technology (ETH), Zurich, Switzerland

    Tony Lomax

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  1. Damien C. Weber
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Correspondence to Damien C. Weber.

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This work has been submitted for oral presentation to the 2014 SIOP meeting, Toronto, Canada.

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Weber, D.C., Ares, C., Malyapa, R. et al. Tumor control and QoL outcomes of very young children with atypical teratoid/rhabdoid Tumor treated with focal only chemo-radiation therapy using pencil beam scanning proton therapy. J Neurooncol 121, 389–397 (2015). https://doi.org/10.1007/s11060-014-1648-2

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