title: 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).
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.
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
authors.forename: M
authors.initials: M
authors.lastname: Benesch
authors.affiliation: Swabian Children's Cancer Center, Children's Hospital Augsburg, Augsburg, Germany.
authors.forename: K
authors.initials: K
authors.lastname: Bartelheim
authors.affiliation: Pediatrics III, University Hospital of Essen, Essen, Germany.
authors.forename: G
authors.initials: G
authors.lastname: Fleischhack
authors.affiliation: Department of Pediatrics, Jena University Hospital, Jena, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Gruhn
authors.affiliation: Department of Pediatric Hematology, Oncology and Neurooncology, University Children's Hospital Würzburg, Würzburg, Germany.
authors.forename: P G
authors.initials: PG
authors.lastname: Schlegel
authors.affiliation: Department of Pediatric Oncology, Hematology, Immunology and Pneumonology, Children's Hospital, University of Heidelberg, Heidelberg, Germany.
authors.forename: O
authors.initials: O
authors.lastname: Witt
authors.affiliation: Children's University Hospital, Friedrich-Alexander University Erlangen-Nürnberg, Erlangen, Germany.
authors.forename: K D
authors.initials: KD
authors.lastname: Stachel
authors.affiliation: Department of Pediatric Hematology and Oncology, Justus Liebig University Gießen, Gießen, Germany.
authors.forename: H
authors.initials: H
authors.lastname: Hauch
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
authors.forename: C
authors.initials: C
authors.lastname: Urban
authors.affiliation: Institute for Medical Statistics, Medical University of Graz, Graz, Austria.
authors.forename: F
authors.initials: F
authors.lastname: Quehenberger
authors.affiliation: Department of Pediatrics, Fondazione IRCCS, Istituto Nazionale Tumori, Milan, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Massimino
authors.affiliation: Institute of Neuropathology, University of Bonn, Bonn, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Pietsch
authors.affiliation: Institute of Neuropathology, University Hospital Münster, Münster, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Hasselblatt
authors.affiliation: 1] Department of Radiological, Oncological and Anatomo-pathological Sciences, University La Sapienza, Rome, Italy [2] IRCCS Neuromed, Pozzilli, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Giangaspero
authors.affiliation: Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
authors.forename: U
authors.initials: U
authors.lastname: Kordes
authors.affiliation: Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Schneppenheim
authors.affiliation: Department of Pediatrics, Semmelweis University, Budapest, Hungary.
authors.forename: P
authors.initials: P
authors.lastname: Hauser
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics, J. W. Goethe University Children's Hospital of Frankfurt, Frankfurt, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.affiliation: 1] Swabian Children's Cancer Center, Children's Hospital Augsburg, Augsburg, Germany [2] Department of Pediatric Hematology and Oncology, University Children's Hospital Münster, Münster, Germany.
authors.forename: M C
authors.initials: MC
authors.lastname: Frühwald
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001706:Biopsy; D016543:Central Nervous System Neoplasms / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009362:Neoplasm Metastasis; D012042:Registries; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*; D033581:Stem Cell Transplantation; D013724:Teratoma / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*
chemical_list: D000970:Antineoplastic Agents