CASE REPORT article
Front. Oncol.
Sec. Pediatric Oncology
Therapy-Related Acute Myeloid Leukemia Following Successful Treatment of High-Risk Neuroblastoma in a Pediatric Patient: A Case Report and Insights into Late Complications
Provisionally accepted
- 1West China Second University Hospital, Sichuan University, Chengdu, China
- 2Sichuan University Key Laboratory of Obstetric and Gynecologic and Pediatric Diseases and Birth Defects, Chengdu, China
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Background: Neuroblastoma is the most common extracranial solid tumor in childhood. With advances in risk-adapted multimodal therapy, survival outcomes for high-risk neuroblastoma have improved substantially. However, prolonged survival has been accompanied by an increasing incidence of therapy-related second malignant neoplasms, which represent a serious late complication and a growing clinical challenge. Case Presentation: We report a rare case of an extremely early-onset secondary malignancy in a young child treated for high-risk neuroblastoma. A 3-year-and-4-month-old girl achieved complete remission after intensive multimodal therapy for stage IV high-risk neuroblastoma. Remarkably, only one month after completion of treatment, she developed therapy-related acute myelomonocytic leukemia (AML, FAB M4 subtype). Bone marrow evaluation revealed high-risk molecular features, including a t(9;11)(p21;q23) translocation resulting in a KMT2A–MLLT3 (MLL/AF9) fusion and concomitant EVI1 overexpression. Results: The patient was treated with intensive AML-directed chemotherapy followed by allogeneic hematopoietic stem cell transplantation. She achieved complete hematologic and molecular remission, with sustained negativity of minimal residual disease. At the last follow-up in July 2025, she remained in continuous remission for 51 months and had returned to normal school life. Conclusion: This case highlights an exceptionally short latency of therapy-related AML as a second malignant neoplasm following modern intensive treatment for high-risk neuroblastoma. It underscores the need for heightened vigilance for secondary malignancies even during the early post-treatment period and emphasizes the importance of long-term surveillance strategies in neuroblastoma survivors. Early recognition and timely allogeneic transplantation may offer curative potential in selected high-risk cases. Despite the overall poor prognosis associated with therapy-related acute myeloid leukemia (t-AML), this patient achieved long-term survival following allogeneic hematopoietic stem cell transplantation, highlighting the potential for successful outcomes even in high-risk cases.
Keywords: allogeneic hematopoietic stem cell transplantation, Genetic predispositions, Neuroblastoma, Secondary neoplasms, therapy-related acute myelomonocytic leukemia
Received: 06 Jan 2026; Accepted: 11 Feb 2026.
Copyright: © 2026 He, Gao, Qiao, He, Guo and Jing. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: XiaoYu Jing
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