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‹ Wed · 20 May 2026
Underserved or high-risk populations

The impact of pre-transplant extramedullary disease on the outcome of allogeneic hematopoietic cell transplantation for acute myeloid leukaemia in children

Extra cancer sites beyond the brain don't worsen survival after bone marrow transplant in children with leukemia, clarifying which patients benefit most from transplant.

In 958 children with AML undergoing allogeneic HCT through EBMT, those with extramedullary disease beyond CNS had a 45% higher relapse incidence compared to bone marrow-only disease, but EMD presence did not significantly impact leukemia-free or overall survival. These registry findings help clarify risk stratification for pediatric AML patients with EMD considered for transplant.

What the study was

Study design
Retrospective cohort study using EBMT registry (PDWP/EBMT)
Population
Children with AML undergoing non-TBI conditioning HCT; EBMT registry
Sample size
958
Category
Treatment Innovation
Maturity
Validated
Journal
Bone Marrow Transplant

Why it surfaced

Large EBMT registry study (n=958) providing important prognostic data for pediatric AML with EMD at HCT. Retrospective design tempers novelty; high population relevance for a vulnerable pediatric oncology subgroup.

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