Details

Autor(en) / Beteiligte

• Benesch, M
• Bartelheim, K
• Fleischhack, G
• Gruhn, B
• Schlegel, P G
• Witt, O
• Stachel, K D
• Hauch, H
• Urban, C
• Quehenberger, F
• Massimino, M
• Pietsch, T
• Hasselblatt, M
• Giangaspero, F
• Kordes, U
• Schneppenheim, R
• Hauser, P
• Klingebiel, T
• Frühwald, M C

Titel

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)

Ist Teil von

• Bone marrow transplantation (Basingstoke), 2014-03, Vol.49 (3), p.370-375

Ort / Verlag

London: Nature Publishing Group UK

Erscheinungsjahr

2014

Quelle

MEDLINE

Beschreibungen/Notizen

• 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.