Department of Pediatrics, CARTOX Program, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Pediatrics, Blood and Marrow Transplantation Program, Keck School of Medicine, University of Southern California, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Nat Rev Clin Oncol. 2021 Jul;18(7):435-453. doi: 10.1038/s41571-021-00474-4. Epub 2021 Feb 19.
Cancer immunotherapies are associated with remarkable therapeutic response rates but also with unique and severe toxicities, which potentially result in rapid deterioration in health. The number of clinical applications for novel immune effector-cell therapies, including chimeric antigen receptor (CAR)-expressing cells, and other immunotherapies, such as immune-checkpoint inhibitors, is increasing. In this Consensus Statement, members of the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network Hematopoietic Cell Transplantation-Cancer Immunotherapy (HCT-CI) Subgroup, Paediatric Diseases Working Party (PDWP) of the European Society of Blood and Marrow Transplantation (EBMT), Supportive Care Committee of the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) and MD Anderson Cancer Center CAR T Cell Therapy-Associated Toxicity (CARTOX) Program collaborated to provide updated comprehensive recommendations for the care of children, adolescents and young adults receiving cancer immunotherapies. With these recommendations, we address emerging toxicity mitigation strategies, we advocate for the characterization of baseline organ function according to age and discipline-specific criteria, we recommend early critical care assessment when indicated, with consideration of reversibility of underlying pathology (instead of organ failure scores) to guide critical care interventions, and we call for researchers, regulatory agencies and sponsors to support and facilitate early inclusion of young patients with cancer in well-designed clinical trials.
癌症免疫疗法具有显著的治疗反应率,但也具有独特且严重的毒性,这可能导致健康状况迅速恶化。新型免疫效应细胞疗法的临床应用数量不断增加,包括嵌合抗原受体 (CAR) 表达细胞,以及其他免疫疗法,如免疫检查点抑制剂。在这份共识声明中,儿科急性肺损伤和脓毒症研究人员 (PALISI) 网络造血细胞移植-癌症免疫治疗 (HCT-CI) 小组、欧洲血液和骨髓移植学会 (EBMT) 的儿科疾病工作组 (PDWP)、儿科移植和细胞治疗联盟 (PTCTC) 的支持护理委员会和 MD 安德森癌症中心 CAR T 细胞治疗相关毒性 (CARTOX) 计划的成员合作,为接受癌症免疫疗法的儿童、青少年和年轻人提供了更新的全面护理建议。通过这些建议,我们解决了新出现的毒性缓解策略,倡导根据年龄和特定于学科的标准来描述基线器官功能,我们建议在需要时早期进行重症监护评估,并考虑潜在病理的可逆性(而不是器官衰竭评分)来指导重症监护干预,我们呼吁研究人员、监管机构和赞助商支持和促进将患有癌症的年轻患者早期纳入精心设计的临床试验中。