The impact of myeloid sarcoma (MS) on clinical outcome of pediatric acute myeloid leukemia (AML) patients remains controversial. Moreover, little is known about the role of stem cell transplantation (SCT) in such patients.
Clinical data of patients with AML under 18 years of age were retrieved from the TARGET dataset. We analyzed the prevalence, clinical profile, molecular characteristics, and prognosis of MS in these patients.
Among 884 pediatric patients with AML, the frequency of MS was 12.3%. Pediatric AML with MS was associated with age under 1-year, abnormal cytogenetics, and KMT2A rearrangement. Moreover, MS was associated with a low complete remission rate, high induction death, poor 5-year EFS, and OS. KMT2A rearrangement had a negative impact on clinical outcome in AML patients with MS. In addition, SCT had no significant effect on the survival of AML patients with MS. Multivariate analysis revealed that MS was an unfavorable prognostic factor in pediatric AML in terms of EFS (Hazard ratio 1.670, P < 0.001) and OS (Hazard ratio 1.623, P = 0.004).
The presence of MS at diagnosis of pediatric AML is associated with poor clinical outcomes, particularly when associated with KMT2A rearrangements. Moreover, pediatric patients with AML and MS may not benefit from SCT.
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The datasets generated and/or analyzed during the current study are available from the TARGET website: https://ocg.cancer.gov/programs/target/data-matrix.
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We would like to thank all study participants and the TARGET group for making these data publicly available.
This work was supported by the National Natural Science Foundation of China (81570140 to J.P. Fang), Guangdong Science and Technology Department (2017B030314026), Natural Science Foundation of Guangdong Province, China (2018A030313680 to L.H. Xu) and the China Scholarship Council [201806385032 to L.H. Xu].
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Xu, L., Wang, Y., Chen, Z. et al. Myeloid sarcoma is associated with poor clinical outcome in pediatric patients with acute myeloid leukemia. J Cancer Res Clin Oncol (2020) doi:10.1007/s00432-020-03128-7
- Acute myeloid leukemia
- Myeloid sarcoma
- Pediatric patients
- Clinical outcome