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Pediatric Acute Myeloid Leukemia (AML) presents significant treatment challenges, particularly post-transplant relapses. The use of the only specific immunotherapy for AML may increase transplant-related toxicities due to the calicheamicin. This scarcity of effective post-transplant strategies underscores a critical need for innovative approaches to improve survival, especially for those undergoing transplantation with active disease. The objective of his study is to describe the experience and effectiveness of the tailored post-transplant maintenance therapy used in pediatric AML, particularly those who entered transplantation with active disease, to enhance survival and cure rates. Methods: Retrospective analysis of 30 pediatric AML patients transplanted between 2014 and 2024. From 2021 on, high risk or FLT3-ITD+ patients transplanted with active disease, received azacytidine or decitabine, FLT3 inhibitors and venetoclax. We collected demographic data, disease characteristics, treatment regimens, and clinical outcomes, using Kaplan-Meier survival estimates and log-rank test to assess the impact of maintenance therapy. Results: The median age of participants was 4 years, 63% male. Most conditioning regimens were myeloablative and busulfan-based (63%). Twenty transplants were haploidentical (67%). Notably, 53% of the transplants were performed with > 5% marrow blasts. Among the total of 30 transplants, due to insurance restraints, only 8 (27%) received post-transplant maintenance therapy: 7 a hypomethylating agent, 5 in combination with venetoclax and 2 FLT3 inhibitors. Maintenance was started at a median of D+59 with a duration of 5 months (1-18 months). Of the 11 patients transplanted with negative MRD, 9 did not receive maintenance and 3 relapsed (33%). Two MRD negative patients received maintenance and remain in remission. Of the 19 patients with active disease or positive MRD, those who received maintenance had a much lower relapse rate (17% vs. 46%). No patient who received maintenance developed poor graft function or graft failure. There was no mortality from infectious causes in this group. The overall survival, relapse-free survival, and median follow-up time for who received maintenance therapy were 86%, 86% and 26 months, compared to 25%, 19% and 42 months for those who did not (p 0.02 and p 0.01 respectively). Conclusion: Preliminary results suggest that maintenance therapy post-transplant significantly enhances outcomes for children with AML, even those with traditionally poorer prognoses due to active disease at the time of transplant. These findings highlight the potential of tailored maintenance therapies to extend survival and reduce relapse rates, providing a new avenue for treatment protocols in a challenging patient subset. Further research is warranted to confirm these findings and potentially guide practice. |
