Tandem Conditioning-Based Myeloablative Hematopoietic Stem Cell Transplantation for Pediatric Patients with Refractory Myeloid Leukemias.
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Survival outcomes of pediatric patients with refractory myeloid leukemias who undergo allogeneic hematopoietic stem cell transplantation (HSCT) remain poor. We investigated the safety and efficacy of a tandem conditioning-based myeloablative HSCT for these high-risk patients. We retrospectively analyzed 10 patients with refractory myeloid leukemias (acute myeloid leukemia, n = 9; juvenile myelomonocytic leukemia blast phase, n = 1), who underwent HSCT following tandem conditioning between 2021 and 2024. All patients were in nonremission at the time of transplantation. Tandem conditioning consisted of FLAG (fludarabine, cytarabine, and granulocyte-colony stimulating factor), followed by myeloablative busulfan and melphalan. Neutrophil and platelet engraftment were observed in 10 patients at a median of 18 days and in 8 patients at a median of 92 days post-transplantation, respectively. All but 1 patient achieved complete remission at the initial post-transplant evaluation performed at a mean of 52 days after HSCT (range, 33 to 91 days). With a median follow-up of 23.2 months for surviving patients, the estimated 2-year leukemia-free survival rate was 70.0 ± 14.5% (n = 7 of 10). The 100- day cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) and the 1-year cumulative incidence of chronic GVHD were 50.0 ± 17.3% and 40.0 ± 17.2%, respectively. Significant early post-HSCT complications included cytomegalovirus DNAemia (n = 7), veno-occlusive disease (n = 4), and hemorrhagic cystitis (n = 4). Although post-HSCT complications were significant, tandem conditioning-based myeloablative HSCT demonstrated promising remission and survival rates in pediatric patients with refractory myeloid leukemias.