Iyer Prasad
Children's Blood and Cancer Centre, KK Women's and Children's Hospital, Singapore.
Duke NUS Medical School, Singapore.
Pediatr Hematol Oncol. 2025 Mar;42(2):126-145. doi: 10.1080/08880018.2025.2453861. Epub 2025 Jan 31.
Pediatric acute myeloid leukemia (AML) is a heterogeneous and aggressive hematological malignancy. Despite advances in treatment, the survival rates remain unsatisfactory, emphasizing the need for innovative therapeutic approaches. This narrative review presents a comprehensive overview of the current approach and likely future directions for pediatric AML. The distinct genetic, epigenetic, and molecular features of pediatric AML contribute to its complex pathophysiology and impact on prognosis. Current treatment practices involve a multifaceted approach combining chemotherapy, molecularly targeted therapies, and hematopoietic stem cell transplantation. However, intensive treatment often leads to significant acute and long-term toxicity. Emerging strategies, including precision medicine, immunotherapy, and novel agents, hold promise for improving outcomes and minimizing adverse effects. Ongoing clinical trials are investigating the potential of these innovative approaches to transform pediatric AML care. By highlighting the evolving treatment paradigms and future perspectives, this review underscores the importance of continued research and development in pediatric AML to enhance the survival rates and quality of life of these young patients.
小儿急性髓系白血病（AML）是一种异质性且侵袭性的血液恶性肿瘤。尽管治疗取得了进展，但生存率仍不尽人意，这凸显了创新治疗方法的必要性。本叙述性综述全面概述了小儿AML的当前治疗方法及可能的未来发展方向。小儿AML独特的遗传、表观遗传和分子特征导致其复杂的病理生理学并影响预后。当前的治疗方法包括化疗、分子靶向治疗和造血干细胞移植相结合的多方面方法。然而，强化治疗往往会导致严重的急性和长期毒性。包括精准医学、免疫疗法和新型药物在内的新兴策略有望改善治疗效果并将不良反应降至最低。正在进行的临床试验正在研究这些创新方法改变小儿AML治疗的潜力。通过强调不断演变的治疗模式和未来前景，本综述强调了在小儿AML领域持续研发以提高这些年轻患者的生存率和生活质量的重要性。
