The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40–50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children’s Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript.
过去几十年间，儿童急性髓系白血病（AML）的预后已逐步改善。然而，复发患者的总体生存率（OS）约为40-50%，化疗难治性患者的生存率更低。这些患儿亟需有效且毒性更低的治疗方案。儿童急性白血病（PedAL）项目是一项战略性全球倡议，旨在克服治疗儿童复发/难治性急性白血病的障碍，该项目由白血病和淋巴瘤协会联合儿童肿瘤协作组、儿童癌症创新疗法联盟以及欧洲儿童急性白血病（EuPAL）基金会等机构共同支持。在欧洲，该研究被设计为一项采用分层方法的复杂临床试验，根据复发患者特征将其分配至靶向抑制剂亚组试验，并在各亚组试验中采用统一的疗效与安全性评估标准。PedAL/EuPAL国际合作项目旨在通过基于生物学的治疗分层标志物，确立首次与二次复发AML的新治疗标准，并为推动药物进入儿童AML一线治疗方案提供关键数据。本文概述了儿童AML的潜在治疗靶点，重点介绍了计划纳入PedAL/EuPAL项目的药物。
肿瘤(癌症)患者之家