Thiotepa and melphalan based single, tandem, and triple high dose therapy and autologous stem cell transplantation for high risk neuroblastoma
Pediatric Blood & Cancer,  Clinical Article

Saarinen–Pihkala UM et al. – Thiotepa– and melphalan based high–dose (HD) regimens, with or without total body irradiation (TBI), appeared to give an outcome comparable to major neuroblastoma (NBL) study groups with acceptable toxicity. Tandem HD therapy gave a 5–year event–free survival (EFS) of 73%, whereas a third HD consolidation did not offer any additional advantage for ultra high risk patients with slow response.

Methods
  • Thirty six children with high risk NBL, diagnosed 1995-2010, had intensive induction and surgery, and were stratified to single, tandem or triple HD-therapy and ASCT, followed by local irradiation and cis-retinoic acid.
  • In inoperable patients surgery was facilitated by preoperative HD-melphalan.
  • Long-term outcome of the old cohort from 1987-1994 was updated.

Results
  • Ten year event-free survival (EFS) from diagnosis was 0.44+/-0.10 of the old and 0.43+/-0.085 of the new cohort.
  • EFS from the last ASCT was 0.53 +/-0.12 and 0.48+/-0.091, respectively.
  • Preoperative HD-melphalan rendered 73% of bulky primaries operable in the new cohort.
  • The 5-yr EFS from ASCT was 0.46+/-0.15 for single and 0.73+/-0.15 for tandem ASCT (P=0.19).
  • All triple ASCT patients, selected by poor/slow response, relapsed or died.

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