Graff Zachary, Burke Michael J, Gossai Nathan
Division of Pediatric Hematology-Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin.
Cancer and Blood Disorders, Children's Minnesota, Minneapolis, Minnesota, USA.
Curr Opin Pediatr. 2024 Feb 1;36(1):64-70. doi: 10.1097/MOP.0000000000001316. Epub 2023 Nov 22.
This review summarizes the current novel therapy landscape in pediatric acute lymphoblastic leukemia (ALL), with a focus on key clinical trials which will shape the future direction of care for these children.
Recent landmark immunotherapy trials in B-ALL have demonstrated significant benefit for children, adolescents, and young adults with relapsed/refractory high-risk leukemia. Due to these successes, current trials are asking the question as to whether immunotherapy can be successfully incorporated upfront. Additionally, therapies targeting novel antigens or molecular pathways are being developed, providing new options for children previously thought to have incurable leukemia.
As survival for ALL has relatively plateaued with maximizing intensity through conventional chemotherapy, continued preclinical and clinical study of novel immunotherapeutic and targeted agents is crucial to further improve outcomes in childhood leukemia.
本综述总结了小儿急性淋巴细胞白血病（ALL）当前的新型治疗格局，重点关注将塑造这些儿童未来治疗方向的关键临床试验。
近期B-ALL的标志性免疫治疗试验已证明，对于复发/难治性高危白血病的儿童、青少年和年轻成人有显著益处。鉴于这些成功案例，当前的试验正在探讨免疫治疗能否成功地用于一线治疗。此外，针对新型抗原或分子途径的疗法正在研发中，为之前被认为患有无法治愈白血病的儿童提供了新的选择。
由于通过传统化疗使强度最大化后，ALL的生存率相对趋于平稳，继续对新型免疫治疗和靶向药物进行临床前和临床研究对于进一步改善儿童白血病的治疗效果至关重要。
