Patient Selection
- Genetic Abnormalities
- High-risk genetic/molecular lesions (e.g., monosomy 5/del(5q), monosomy 7, high allelic ratio FLT3-ITD mutations): Proceed to hematopoietic stem cell transplantation (HSCT) after achieving first complete remission (CR1).
- Low-risk lesions (e.g., CBF, NPM1, CEBPA bZIP mutations): Avoid HSCT in CR1 even if minimal residual disease (MRD) remains positive after initial induction.
- Treatment Response
- Primary induction failure (persistent morphologic blasts ≥5% after 1–2 induction cycles): Consider early HSCT at induction completion.
- MRD-positive at induction end without additional risk factors: Proceed to HSCT in CR1.
- Relapsed disease: Attempt to achieve second complete remission (CR2) before HSCT.
Preparative Regimens
- Myeloablative Conditioning (MAC)
- Recommended for first or second HSCT in children without comorbidities and with suitable donors.
- Exclude total body irradiation (TBI) from pediatric MAC protocols.
- Reduced-Intensity Conditioning (RIC)
- Consider for children with inherited bone marrow failure syndromes progressing to myelodysplastic syndrome (MDS).
Donor Selection
- Sibling Donors
- Preferred due to lower graft-versus-host disease (GVHD) incidence and superior survival compared to matched/unrelated or umbilical cord blood (UCB) donors.
- Alternative Donors
- UCB recipients: Associated with reduced chronic GVHD but higher treatment-related mortality (TRM), delayed platelet/neutrophil engraftment, and slower immune reconstitution.
- Bone marrow (BM) > unmanipulated peripheral blood (PB): Prioritize BM as the graft source when feasible.
Clinical Implications
HSCT remains a cornerstone in treating pediatric AML. These guidelines aim to improve transplant success, reduce complications, and enhance survival quality. However, clinicians must individualize treatment plans based on patient-specific factors, including comorbidities, disease biology, and donor availability.
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