Department of Pediatrics, Division of Hematology, Oncology, and BMT, Medical College of Wisconsin, Milwaukee, WI.
Department of Pediatric Oncology, Dana-Farber Cancer Institute, Division of Hematology/Oncology, Boston Children's Hospital and Harvard Medical School, Boston, MA.
Am Soc Clin Oncol Educ Book. 2024 Jun;44(3):e438690. doi: 10.1200/EDBK_438690.
Despite improved outcomes in newly diagnosed pediatric AML, relapsed disease remains a therapeutic challenge. Factors contributing to slow progress in improving outcomes include inherent challenges in pediatric clinical trial accrual and the scarcity of novel targeted/immunotherapy agents available for pediatric development. This paradigm is changing, however, as international collaboration grows in parallel with the development of promising targeted agents. In this review, we discuss the therapeutic landscape of relapsed pediatric AML, including conventional chemotherapy, targeted therapies, and the challenges of drug approvals in this patient population. We highlight current efforts to improve communication among academia, industry, and regulatory authorities and discuss the importance of international collaboration to improve access to new therapies. Among the therapeutic options, we highlight the approach to second hematopoietic stem cell transplant (HSCT) and discuss which patients are most likely to benefit from this potentially curative intervention. Importantly, we acknowledge the challenges in providing these high-risk interventions to our patients and their families and the importance of shared communication and decision making when considering early-phase clinical trials and second HSCT.
尽管新诊断的小儿急性髓系白血病（AML）的治疗效果有所改善，但疾病复发仍然是一个治疗挑战。导致治疗效果改善进展缓慢的因素包括儿科临床试验入组方面固有的挑战，以及可供儿科开发的新型靶向/免疫治疗药物稀缺。然而，随着国际合作的发展与有前途的靶向药物的开发齐头并进，这种模式正在发生变化。在这篇综述中，我们讨论了复发型小儿 AML 的治疗现状，包括常规化疗、靶向治疗以及该患者群体药物批准面临的挑战。我们强调了目前在学术界、工业界和监管机构之间加强沟通的努力，并讨论了国际合作对改善新疗法可及性的重要性。在治疗选择中，我们重点介绍了二次造血干细胞移植（HSCT）的方法，并讨论了哪些患者最有可能从这种潜在的治愈性干预中获益。重要的是，我们认识到向我们的患者及其家属提供这些高风险干预措施所面临的挑战，以及在考虑早期临床试验和二次 HSCT 时进行共享沟通和决策的重要性。
