Pediatric Hematology/Oncology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Cell Factory, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Front Immunol. 2020 Oct 15;11:567531. doi: 10.3389/fimmu.2020.567531. eCollection 2020.
Dramatic progress in the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from alternative sources in pediatric patients has been registered over the past decade, providing a chance to cure children and adolescents in need of a transplant. Despite these advances, transplant-related mortality due to infectious complications remains a major problem, principally reflecting the inability of the depressed host immune system to limit infection replication and dissemination. In addition, development of multiple infections, a common occurrence after high-risk allo-HSCT, has important implications for overall survival. Prophylactic and preemptive pharmacotherapy is limited by toxicity and, to some extent, by lack of efficacy in breakthrough infections. T-cell reconstitution is a key requirement for effective infection control after HSCT. Consequently, T-cell immunotherapeutic strategies to boost pathogen-specific immunity may complement or represent an alternative to drug treatments. Pioneering proof of principle studies demonstrated that the administration of donor-derived T cells directed to human herpesviruses, on the basis of viral DNA monitoring, could effectively restore specific immunity and confer protection against viral infections. Since then, the field has evolved with implementation of techniques able to hasten production, allow for selection of specific cell subsets, and target multiple pathogens. This review provides a brief overview of current cellular therapeutic strategies to prevent or treat pathogen-related complications after HSCT, research carried out to increase efficacy and safety, including T-cell production for treatment of infections in patients with virus-naïve donors, results from clinical trials, and future developments to widen adoptive T-cell therapy access in the HSCT setting.
在过去的十年中,异体造血干细胞移植(allo-HSCT)在儿科患者中的替代来源方面取得了显著的成果,为需要移植的儿童和青少年提供了治愈的机会。尽管取得了这些进展,但由于感染并发症导致的移植相关死亡率仍然是一个主要问题,主要反映了受抑制的宿主免疫系统无法限制感染的复制和传播。此外,在高危 allo-HSCT 后,多种感染的发生是常见的,这对总体生存率有重要影响。预防性和先发制人的药物治疗受到毒性的限制,在某种程度上也受到突破性感染疗效的限制。T 细胞重建是 HSCT 后有效控制感染的关键要求。因此,T 细胞免疫治疗策略可以增强针对病原体的免疫,从而补充或替代药物治疗。开创性的原理研究证明,基于病毒 DNA 监测,给予针对人类疱疹病毒的供体衍生 T 细胞,可以有效地恢复特异性免疫,并提供针对病毒感染的保护。从那时起,该领域已经发展起来,实施了能够加速生产、允许选择特定细胞亚群、并靶向多种病原体的技术。本文简要概述了当前用于预防或治疗 HSCT 后与病原体相关的并发症的细胞治疗策略,包括为治疗病毒-naïve 供体患者的感染而进行的提高疗效和安全性的研究、临床试验结果以及未来的发展,以扩大 HSCT 环境中过继性 T 细胞治疗的应用。