Department of Pediatrics, Centro di Ricerca Matilde Tettamanti, San Gerardo Hospital, University of Milano-Bicocca Monza, Italy.
Front Oncol. 2013 Apr 30;3:106. doi: 10.3389/fonc.2013.00106. eCollection 2013.
Despite the survival of pediatric patients affected by hematological malignancies being improved in the last 20 years by chemotherapy and hematopoietic stem cell transplantation, a significant amount of patients still relapses. Treatment intensification is limited by toxic side effects and is constrained by the plateau of efficacy, while the pipeline of new chemotherapeutic drugs is running short. Therefore, novel therapeutic strategies are essential and researchers around the world are testing in clinical trials immune and gene-therapy approaches as second-line treatments. The aim of this review is to give a glance at these novel promising strategies of advanced medicine in the field of pediatric leukemias. Results from clinical protocols using new targeted "smart" drugs, immunotherapy, and gene therapy are summarized, and important considerations regarding the combination of these novel approaches with standard treatments to promote safe and long-term cure are discussed.
尽管过去 20 年来，通过化疗和造血干细胞移植，儿科血液系统恶性肿瘤患者的生存率得到了提高，但仍有大量患者复发。治疗强化受到毒性副作用的限制，并且受到疗效平台的限制，而新的化疗药物的研发线也在枯竭。因此，需要新的治疗策略，世界各地的研究人员正在临床试验中测试免疫和基因治疗方法作为二线治疗。本文旨在概述儿科白血病领域这些有前途的先进医学新策略。总结了使用新型靶向“智能”药物、免疫疗法和基因疗法的临床方案的结果，并讨论了将这些新方法与标准治疗相结合以促进安全和长期治愈的重要考虑因素。
