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

Hsiu Ju Yen, Ting Yen Yu, Chih Ying Lee, Giun Yi Hung, Tzeon Jye Chiou, Hsin Hung Chen, Yi Yen Lee, Muh Lii Liang, Yi Wei Chen

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)148-154
Number of pages7
JournalJournal of the Chinese Medical Association
Volume82
Issue number2
DOIs
Publication statusPublished - Feb 1 2019
Externally publishedYes

Keywords

  • Pediatric embryonal tumors
  • Tandem transplant
  • Thiotepa-based conditioning regimen

ASJC Scopus subject areas

  • Medicine(all)

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