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Multimodal therapy in children and adolescents with newly diagnosed atypical teratoid rhabdoid tumor: individual pooled data analysis and review of the literature

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Abstract

Atypical teratoid rhabdoid tumour (ATRT) is a malignant tumour of the central nervous system with a dismal prognosis. There is no consensus on optimal treatment and different multimodal strategies are currently being used in an attempt to improve outcomes. To evaluate the impact of high-dose chemotherapy followed by autologous stem-cell rescue (HD48 SCR), radiotherapy (RT) at first line, intrathecal chemotherapy (IT) and extent of surgical resection upon recurrence-free survival (RFS) and overall survival (OS). An online database search identified prospective and retrospective studies focused on the treatment of children and adolescents with newly diagnosed ATRT. Clinical, therapeutic and outcome data were extracted and an individual pooled data analysis was conducted. Out of 389 publications, 12 manuscripts were included in our review. Data from 332 patients were analysed. Median age at diagnosis was 37 months (range 1–231). HD-SCR, RT and IT had been administered to 28.6 % (58/203), 49.6 % (118/238) and 21 % (65/310) of the patients, respectively. Gross total resection (GTR) had been achieved in 46.5 % (152/327) of the cases. In the multivariate analysis, hazard ratios (95 % Confidence Interval) for HD-SCR were: RFS-HR = 0.570 (0.357–0.910) p = 0.019, and OS-HR = 0.388 (0.214–0.704) p = 0.002; and for RT: RFS-HR = 0.551 (0.351–0.866) p = 0.01, and OS-HR = 0.393 (0.216–0.712) p = 0.002. IT and GTR were not significantly associated with improved RFS or OS in the multivariate analysis. In our pooled data review, HD-SCR and RT at first line were associated with improved outcomes in children and adolescents with newly diagnosed ATRT.

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    Authors and Affiliations

    1. Children and Young People’s Unit, The Royal Marsden NHS Foundation Trust, Downs Road, Sutton, Surrey, SM2 5PT, UK

      D. Schrey, F. Carceller Lechón, L. Moreno, L. V. Marshall & S. Zacharoulis

    2. Divisions of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, 15 Cotswold Road, Sutton, Surrey, SM2 5NG, UK

      F. Carceller Lechón, L. Moreno, L. V. Marshall & S. Zacharoulis

    3. Department of Surgery, St Mark’s Hospital, Watford Road, Harrow, Middlesex, HA1 3UJ, UK

      G. Malietzis

    4. Department of Surgery and Cancer, Imperial College, Paddington, London, W2 1NY, UK

      G. Malietzis & T. Athanasiou

    5. Clinical Research Programme, Spanish National Cancer Research Centre (CNIO), Calle de Melchor Fernandez Almagro, num. 3, 28029, Madrid, Spain

      L. Moreno

    6. Department of Pediatric and Adolescent Oncology, Institute Gustave Roussy, 114 rue Edouard Vaillant, 94805, Villejuif, France

      C. Dufour

    7. Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, 450 Brookline Ave, Boston, MA, 02215, USA

      S. Chi

    8. Division of Pediatric Hematology Oncology and Bone Marrow Transplantation, Alberta Children’s Hospital, Calgary, AB, T3B 6A8, Canada

      L. Lafay-Cousin

    9. Department for Pediatric Hematology and Oncology, University Hospital Hamburg-Eppendorf, Martinistr. 52, 20246, Hamburg, Germany

      K. von Hoff

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    D. Schrey and F. Carceller Lechón have been contributed equally to this work.

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    Schrey, D., Carceller Lechón, F., Malietzis, G. et al. Multimodal therapy in children and adolescents with newly diagnosed atypical teratoid rhabdoid tumor: individual pooled data analysis and review of the literature. J Neurooncol 126, 81–90 (2016). https://doi.org/10.1007/s11060-015-1904-0

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