Extramedullary leukemia in children with acute myeloid leukemia: A population‐based cohort study from the Nordic Society of Pediatric Hematology and Oncology (NOPHO)

Abstract Background The prognostic significance of extramedullary leukemia (EML) in childhood acute myeloid leukemia is not clarified. Procedure This population‐based study included 315 children from the NOPHO‐AML 2004 trial. Results At diagnosis, 73 (23%) patients had EML: 39 (12%) had myeloid sarcoma, 22 (7%) had central nervous system disease, and 12 (4%) had both. EML was associated with young age (median age: 2.6 years), a high white blood cell count (median: 40 × 10 9 /l), M5 morphology (40%), and 11q23/ MLL ( KMT2A ) rearrangements (34%). No patient received involved field radiotherapy. Five‐year event‐free survival did not differ significantly between the EML and the non‐EML patients (54% vs. 45%, P = 0.57), whereas 5‐year overall survival (OS) was significantly lower in the EML group (64% vs. 73%, P = 0.04). The risk of induction death was significantly higher for EML patients (8% vs. 1%, P = 0.002). There was a trend toward a lower risk of relapse for EML patients (5‐year cumulative incidence of relapse 33% vs. 49%, P = 0.16). Traumatic lumbar puncture did not adversely affect survival in this cohort. Conclusions EML was associated with increased risk of induction death impacting the OS. No patients relapsed at the primary site of the myeloid sarcoma despite management without radiotherapy.