Saultier Paul, Michel Gérard
Department of Pediatric Hematology, Immunology and Oncology, Aix Marseille Université, APHM, INSERM, INRAe, C2VN, La Timone Children's Hospital, Marseille, France.
Department of Pediatric Hematology, Immunology and Oncology, Aix Marseille Université, APHM, CERESS, La Timone Children's Hospital, Marseille, France.
Blood. 2024 May 2;143(18):1795-1806. doi: 10.1182/blood.2023019804.
The population of survivors of childhood leukemia who reach adulthood is growing due to improved therapy. However, survivors are at risk of long-term complications. Comprehensive follow-up programs play a key role in childhood leukemia survivor care. The major determinant of long-term complications is the therapeutic burden accumulated over time. Relapse chemotherapy, central nervous system irradiation, hematopoietic stem cell transplantation, and total body irradiation are associated with greater risk of long-term complications. Other parameters include clinical characteristics such as age and sex as well as environmental, genetic, and socioeconomic factors, which can help stratify the risk of long-term complications and organize follow-up program. Early diagnosis improves the management of several late complications such as anthracycline-related cardiomyopathy, secondary cancers, metabolic syndrome, development defects, and infertility. Total body irradiation is the treatment associated with worse long-term toxicity profile with a wide range of complications. Patients treated with chemotherapy alone are at a lower risk of long-term complications, although the optimal long-term follow-up remains unclear. Novel immunotherapies and targeted therapy are generally associated with a better short-term safety profile but still require careful long-term toxicity monitoring. Advances in understanding genetic susceptibility to long-term complications could enable tailored therapeutic strategies for leukemia treatment and optimized follow-up programs.
由于治疗方法的改进，成年期的儿童白血病幸存者数量正在增加。然而，幸存者面临长期并发症的风险。全面的随访计划在儿童白血病幸存者护理中起着关键作用。长期并发症的主要决定因素是随着时间积累的治疗负担。复发化疗、中枢神经系统照射、造血干细胞移植和全身照射与更高的长期并发症风险相关。其他参数包括年龄和性别等临床特征以及环境、遗传和社会经济因素，这些因素有助于对长期并发症风险进行分层并组织随访计划。早期诊断有助于改善几种晚期并发症的管理，如蒽环类药物相关的心肌病、继发性癌症、代谢综合征、发育缺陷和不孕症。全身照射是与更差的长期毒性特征相关的治疗方法，会引发多种并发症。仅接受化疗的患者长期并发症风险较低，尽管最佳的长期随访仍不明确。新型免疫疗法和靶向疗法通常具有较好的短期安全性，但仍需要仔细进行长期毒性监测。对长期并发症遗传易感性认识的进展可能使白血病治疗的定制治疗策略和优化的随访计划成为可能。
