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. 2014 Mar;49(3):370-5. doi: 10.1038/bmt.2013.208. Epub 2014 Jan 13.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Biopsy
  • Central Nervous System Neoplasms / drug therapy
  • Central Nervous System Neoplasms / surgery
  • Central Nervous System Neoplasms / therapy*
  • Child, Preschool
  • Combined Modality Therapy
  • Disease Progression
  • Disease-Free Survival
  • Europe
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Neoplasm Metastasis
  • Registries
  • Retrospective Studies
  • Rhabdoid Tumor / drug therapy
  • Rhabdoid Tumor / surgery
  • Rhabdoid Tumor / therapy*
  • Stem Cell Transplantation*
  • Teratoma / drug therapy
  • Teratoma / surgery
  • Teratoma / therapy*

Substances

  • Antineoplastic Agents