Reduced-toxicity conditioning regimen with busulfan, fludarabine, rATG, and 400 cGy TBI in pediatric patients undergoing hematopoietic stem cell transplant for high-risk hematologic malignancies

Jenna Rossoff*, David Jacobsohn, Soyang Kwon, Morris Kletzel, Reggie E Duerst, William T Tse, Jennifer Schneiderman, Sonali Chaudhury

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Myeloablative conditioning regimens decrease the risk of relapse in pediatric patients undergoing allogeneic hematopoietic stem cell transplant (HCT) for hematologic malignancies, but cause significant toxicities. Procedure: This prospective study evaluated the use of a reduced-toxicity, myeloablative regimen with dose-adjusted busulfan, fludarabine, antithymocyte globulin and 400 cGy of total body irradiation in 40 patients < 21 years of age undergoing HCT for high-risk leukemias. Busulfan pharmacokinetics were measured to target 4000 μmol*min/day in the first 30 patients; this was increased to 5000 μmol*min/day in the subsequent 10 in efforts to further decrease relapse risk. Results: Overall survival at two- and five-years post-HCT was 67% and 51%, respectively. Relapse occurred in 11 patients (28%) at a median of seven months and was the leading cause of death. Transplant-related mortality was 8% and 13% at 100 days and one-year post-HCT, respectively. Trends toward improved survival were seen in patients transplanted for myeloid disease using bone marrow as stem cell source who achieved a busulfan AUC > 4000 μmol*min/day with two-year relapse-free survival approaching 80%. Conclusions: This conditioning regimen is safe and effective in patients with high-risk leukemias, particularly myeloid disease. Larger studies are needed to compare its safety and efficacy to other myeloablative regimens in this population.

Original languageEnglish (US)
Article numbere29087
JournalPediatric Blood and Cancer
Volume68
Issue number8
DOIs
StatePublished - Aug 2021

Keywords

  • pediatric leukemia
  • reduced-toxicity conditioning regimen
  • stem cell transplant

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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