Therapy-related acute myeloid leukemia following aggressive treatment for pediatric medulloblastoma: a case report of a 13-year-old and review of literature.
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INTRODUCTION AND IMPORTANCE: Medulloblastoma (MBL) is the most common malignant brain tumor in children, accounting for up to 25% of primary central nervous system neoplasms. Standard treatment includes surgical resection, chemotherapy, and craniospinal irradiation (CSI). While these multimodal therapies have significantly improved survival, they are associated with long-term toxicities. One rare but serious complication is therapy-related acute myeloid leukemia (t-AML), which results from exposure to cytotoxic chemotherapy and/or radiotherapy. t-AML is known for its poor prognosis, low treatment response, and increased toxicity, making it a particularly challenging outcome in pediatric oncology. CASE PRESENTATION: We present the case of a pediatric patient with medulloblastoma who experienced three disease relapses. During treatment with etoposide for recurrent medulloblastoma, the patient developed t-AML. Despite the typically poor outcomes associated with t-AML, the patient achieved remission from leukemia. However, medulloblastoma remained refractory. CLINICAL DISCUSSION: This case highlights the dual burden of disease recurrence and secondary malignancy in pediatric oncology patients. The development of t-AML following etoposide therapy raises important considerations about cumulative treatment-related risks. While remission of t-AML was successfully achieved, the ongoing progression of medulloblastoma underscores the complexity of managing patients with multiple, concurrent oncologic challenges. It also calls attention to the limitations of current therapeutic approaches in preventing and treating long-term complications. CONCLUSION: The case underscores the importance of vigilant long-term follow-up and surveillance for hematologic abnormalities in medulloblastoma patients, especially those exposed to high-risk chemotherapeutic agents. Early detection and intervention for therapy-related complications like t-AML are essential to optimize care, even as the primary malignancy may continue to progress.