KEY TAKEAWAYS
- The study aimed to investigate the prognostic impact of EMI at diagnosis and the effects of SCT and GO treatment in patients with pediatric AML.
- Researchers found that EMI at diagnosis is an adverse prognostic factor in pediatric AML, with GO treatment potentially improving survival; further investigation is ongoing.
The outcome of extramedullary infiltration (EMI) in pediatric acute myeloid leukemia (AML) is controversial, and little is known about the implications of stem cell transplantation (SCT) and gemtuzumab ozogamicin (GO) treatment on patients with EMI.
Weiya Li and the team aimed to evaluate the prognostic significance of EMI at diagnosis in pediatric AML and to examine the impact of SCT and GO treatment on the survival outcomes of these patients.
They performed an inclusive analysis of clinical data from 713 pediatric patients with AML, obtained from the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) dataset. They analyzed the clinical and prognostic characteristics of patients with EMI at diagnosis and relapse, focusing on the impact of SCT and GO treatment on survival outcomes.
About 123 patients were identified to have EMI at diagnosis and 64 presented with EMI at relapse. The presence of EMI was associated with age ≤2 years, M5 morphology, abnormal karyotype, and KMT2A rearrangements. Hyperleukocytosis and complex karyotype were more prevalent in patients with EMI at relapse. Additionally, patients with EMI at diagnosis had a reduced incidence of FLT3 ITD-/NPM1+, whereas those with EMI at relapse displayed a lower frequency of FLT3 ITD+.
Patients with EMI at diagnosis exhibited a lower complete remission (CR) rate at the end of Induction Course 1 and higher relapse incidence. Importantly, EMI at diagnosis independently predicted both shorter event-free survival (EFS) and overall survival (OS). Regarding relapse patients, the occurrence of EMI at relapse showed no impact on OS. However, relapse patients with myeloid sarcoma (MS)/no central nervous system (CNS) exhibited poorer OS compared to those with CNS/no MS. Furthermore, regarding patients with EMI at diagnosis, SCT failed to improve the survival, whereas GO treatment potentially enhanced OS.
The study concluded that EMI at diagnosis is an independent adverse prognostic risk factor for pediatric AML and that GO treatment potentially improves survival for patients with EMI at diagnosis.
No funding information was provided.
Source: https://pubmed.ncbi.nlm.nih.gov/38644612/
Li W, Shi M, Zhou P, et al. (2024). “Extramedullary infiltration in pediatric acute myeloid leukemia: Results from the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) initiative.” Pediatr Blood Cancer. 2024 Jul;71(7):e31014. doi: 10.1002/pbc.31014. Epub 2024 Apr 21. PMID: 38644612.
