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 (2014) 49, 370375 & 2014 Macmillan Publishers Limited All rights reserved 0268-3369/14

http://www.nature.com/bmt

Web End =www.nature.com/bmt

ORIGINAL ARTICLE

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 Benesch1, K Bartelheim2, G Fleischhack3, B Gruhn4, PG Schlegel5, O Witt6, KD Stachel7, H Hauch8, C Urban1,F Quehenberger9, M Massimino10, T Pietsch11, M Hasselblatt12, F Giangaspero13,14, U Kordes15, R Schneppenheim15, P Hauser16, T Klingebiel17 and MC Frhwald2,18

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 identied. Nine patients presented with metastatic disease at

diagnosis. A partial or subtotal resection was achieved in 11, a total resection in ve and a biopsy in three patients. Patients received a median of six chemotherapy cycles prior to HDCT. Additional radiotherapy was performed in 14 patients (rst-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 (rst 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 benet from HDCT with radiotherapy. The denitive impact of this treatment modality has to be evaluated prospectively in a randomized trial.

Bone Marrow Transplantation (2014) 49, 370375; doi:http://dx.doi.org/10.1038/bmt.2013.208

Web End =10.1038/bmt.2013.208 ; published online 13 January 2014

Keywords: atypical teratoid/rhabdoid tumors; auto-SCT; children

INTRODUCTIONAtypical teratoid/rhabdoid tumors (AT/RT) are highly malignant tumors of the central nervous system (CNS).1 Among children below the age of 3 years, AT/RT constitute the most common malignant CNS tumors (17.3%), followed by medulloblastomas (16%) and primitive neuroectodermal tumors of the CNS (13.3%).2 In a population-based study, the age-standardized incidence rate of AT/RT was calculated at 1.38 per 1 000 000 personyears.2 The median age...