Superior long-term survival with acceptable safety of ATG/G-CSF–based haplo-HSCT with intensified BU+MEL/TT conditioning in CR Pediatric Non-DS–AMKL
By
Fei Pan
Jing Long
Xing-yu Cao
Yue Lu
Jian-ping Zhang
Yan-li Zhao
Min Xiong
Fang-min Pan
Guan-lan Yue
Kang Gao
Zhi-jie Wei
April 30, 2026
Clinical Scorecard: Enhanced Long-Term Survival and Acceptable Safety Profile of ATG/G-CSF-Based Haplo-HSCT with Intensified BU+MEL/TT Conditioning in Pediatric Non-DS–AMKL in First Complete Remission
At a Glance
Category Detail
Condition Non-Down syndrome acute megakaryoblastic leukemia (non-DS-AMKL) Key Mechanisms Intensified conditioning regimens improve overall survival (OS) and leukemia-free survival (LFS) without increasing early non-relapse mortality (NRM). Target Population Pediatric patients with non-DS-AMKL in first complete remission (CR1). Care Setting Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in a clinical setting.
Key Highlights
Intensified BU+MEL/TT conditioning improved 5-year OS (75.2% vs 50.0%) and LFS (76.3% vs 50.0%) compared to standard BU/CY. No significant differences in early TRM, engraftment, GVHD, or viral reactivations between conditioning groups. Pre-transplant NR/PR status significantly increased relapse and NRM, highlighting the importance of achieving remission before transplantation. Guideline-Based Recommendations
Diagnosis
Confirm diagnosis of non-DS-AMKL according to the 2022 WHO classification. Management
Consider intensified conditioning regimens (BU+MEL/TT) for patients in CR1 to improve survival outcomes. Monitoring & Follow-up
Monitor for relapse and non-relapse mortality post-transplant, especially in patients with NR/PR status. Risks
Complex cytogenetics and pre-transplant NR/PR status are independent adverse factors affecting OS and LFS. Patient & Prescribing Data
Pediatric patients aged <14 years diagnosed with non-DS-AMKL.
Haplo-HSCT with intensified conditioning shows promising outcomes without increasing early complications.
Clinical Best Practices
Achieve remission before transplantation to improve outcomes. Utilize haplo-HSCT as a viable donor option for high-risk pediatric AML. References
