Which children and young adults with acute myeloid leukaemia should receive transplant in first remission? This episode explores a landmark 2025 analysis from the Children's Oncology Group that revisits this longstanding question through a contemporary molecular lens.

Huang and colleagues, publishing in the Journal of Clinical Oncology, analysed outcomes for over 1,400 patients from the AAML0531 and AAML1031 trials—studies that enrolled patients up to age 29. By retrospectively applying contemporary molecular risk criteria from the current AAML1831 trial, including newly recognised adverse genetic lesions and measurable residual disease thresholds, they revealed striking patterns in transplant benefit across redefined risk groups.

We unpack how this molecular reclassification informs our understanding of transplant outcomes, from the substantial improvements in disease-free survival for most high-risk patients to the unexpected findings in certain subgroups like monosomy 7. Along the way, we explore what these retrospective insights might mean for clinical practice in the genomic era of AML management—from approaches to molecular profiling at diagnosis to considerations around transplant timing in first remission.

Episode type: Deep Dive—intended for leukaemia and transplant health care professionals or scientists involved in AML diagnostics or research.

Reference: Hematopoietic Stem Cell Transplantation Outcomes for High-Risk AML: A Report From the Children’s Oncology Group by Huang BJ et al., Journal of Clinical Oncology, 2025. DOI: 10.1200/JCO-24-01841