The impact on outcomes by using thiotepa in tandem transplant for pediatric high-risk embryonal brain tumors.

Background: Despite aggressive treatment including surgery, radiotherapy, and adjuvant chemotherapy, the outcome of pediatric high-risk embryonal brain tumors remains poor; especially in young children, in whom early radiotherapy inevitably brings significant long-term morbidities. Single or tandem autologous stem cell transplant has been reported to improve outcomes; but optimal use is not well defined.

Methods: Pediatric patients with high-risk embryonal brain tumors who underwent tandem transplant as consolidation from August 2011 to December 2017 were included. We performed a retrospective chart review and analyzed the outcomes to identify possible prognostic factors.

Results: Eleven pediatric patients with high-risk embryonal brain tumors were enrolled. They received double or triple autologous transplant at complete response in 5 patients and at partial response in 6 for a total of 24 transplants. There were five atypical teratoid rhabdoid tumors, four medulloblastoma, one primitive neuroectodermal tumors, and one pineoblastoma. Median age at diagnosis was 1.8 years (range, 0.6-11.2 years) and at transplant was 2.2 years (range, 1.2-11.9 years). Thiotepa-based regimens were used in 13 cycles of conditioning. All patients achieved successful engraftment. No transplant-related mortality was identified. With a median follow-up of 21.2 months (range, 6.9-51.8 months), seven patients had disease progression. Disease entity and the use of one or more cycles of thiotepa-based regimen during tandem transplant had statistically significant impact on both progression-free survival and overall survival.

Conclusion: With successful engraftment and manageable toxicity, tandem transplant in pediatric patients with high-risk embryonal brain tumor is feasible and safe. Patients receiving tandem transplant with one or more cycles of thiotepa-based regimen might have better outcome than those without. In combination with salvage radiotherapy, a favorable 2-year overall survival could be achieved in the majority of patients.